1800 620 420
Close menu

Daratumumab – first new myeloma agent PBAC-recommended in years

Daratumumab – first new myeloma agent PBAC-recommended in years

For the first time in 13 years, a new agent with a different mode of action against myeloma, daratumumab (Darzalex®), has been recommended for Pharmaceutical Benefits Scheme (PBS) reimbursement.

At the July 2020 meeting of the Pharmaceutical Benefits Advisory Committee (PBAC), daratumumab received a positive recommendation for PBS listing as a second line treatment, in combination with bortezomib (Velcade®) and dexamethasone for patients with myeloma.

In a submission to the PBAC in June 2020, the Leukaemia Foundation provided consumer comments in relation to daratumumab, based on the everyday experiences of people living with myeloma.

Peter, a myeloma patient from Western Australia, said:

Darzalex “This drug is proving revolutionary in the fight against this terminal blood cancer and should be available as a permanent line of therapy [as] it can extend or save lives.”

The Leukaemia Foundation’s submission also stated that, based on the summary clinical trial information provided, “we understand daratumumab-based combinations have demonstrated efficacy in comparison to other agents tested in head-to-head relapsed or refractory myeloma trials in terms of delaying disease progression and response rates, and have also been shown to lead to molecular remissions in a greater number of patients with relapsed multiple myeloma”.

The PBAC noted in its recommendation that daratumumab monotherapy would be provided by the sponsor (Janssen), on a compassionate basis, to all eligible relapsed and/or refractory (R/R) myeloma patients who have no other PBS-funded treatment options.

A study, published in an August 2020 online issue of Journal of Hematology & Oncology, confirmed and extended the previously known effectiveness and tolerability of daratumumab plus standard of care (bortezomib/dexamethasone) as a treatment for R/R myeloma.

The Leukaemia Foundation also made a submission to the PBAC for carfilzomib (Kyprolis®).

“We received eight responses to the request for feedback on this submission,” said Emily Forrest, the Leukaemia Foundation’s Head of Policy and Advocacy.

“Views amongst patients and their families/carers were mixed, with some concerned about the side-effects of the treatment,” said Emily.

“There is a high unmet need for new treatments for myeloma, which is currently an incurable disease which becomes progressively harder to treat after each relapse as patients become refractory to different treatments.

“We believe that the listing amendment for carfilzomib would provide clinicians with additional choice in therapy regimens for patients with refractory myeloma.”

Carfilzomib also received a positive recommendation to amendments to the dosing regimen and a change to a streamlined authority level, as sought by the sponsor (Amgen Australia).

Emily said the Leukaemia Foundation would provide submissions to the PBAC for consideration at its November 2020 meeting for elotuzumab (Emplicit®) and ixazomib (Ninlaro®) for patients with R/R myeloma.

Read Shirley Irwin’s experience with myeloma and a range of treatments including carfilzomib and daratumumab. (add link to story)

Shirley’s “so well” on her latest treatment – daratumumab

Shirley’s “so well” on her latest treatment – daratumumab

Shirley and her three grandsons
Shirley and her three grandsons, Reuben, Darcy and Finn, with the fox patchworks they made together

Shirley Irwin has had radiotherapy and two transplants, she’s dipped into savings to access treatment, had surgery, five different lines of treatment, and relapsed twice.

Despite all that has happened since her diagnosis with high-risk myeloma in May 2018, Shirley says, “I would say I have had a milder journey”.

Now on the monoclonal antibody, daratumumab (Darzalex®), which she started in July 2020, Shirley doesn’t know what her next option will be, but she’s prepared to personally pay for access to new therapies again, if they are not available on the Pharmaceutical Benefits Scheme (PBS). She and her husband, Bruce*, have a second property that they are prepared to sell, if necessary.

Now though, Shirley says she’s “so well, feels so good and is absolutely normal except, except weak to walk”. The daratumumab is working for her, she has no side-effects and hopes to continue this treatment on an ongoing monthly basis.

“If I could coast along like this, it would be lovely,” said Shirley.

Myeloma diagnosis and initial treatment

Myeloma came out of nowhere for Shirley, appearing as a sudden pain “that was scary”, while on holiday. She and Bruce had been away for six weeks in their caravan, had met up with friends from Perth, and were at Hall’s Gap.

They continued down the coast and up to Canberra before Shirley decided the pain was too great to go any further.

“It was leaving me uncomfortable and affecting my body. I said to Bruce, ‘I want to go home, straight home’,” said Shirley about returning to Ocean Grove, the seaside Victorian town where they have lived all their married life – 45 years.

She went to the doctor and after some tests a lesion was found on her spine and she was told she had high-risk myeloma. She was 69 and knew nothing about myeloma.

“I’d never had surgery, I’d never been ill; it was a big learning curve,” said Shirley, now 71.

Treatment began immediately. Radiotherapy on her spine, to shrink the lesion, before induction treatment with bortezomib (Velcade®) and a stem cell harvest, in preparation for an autologous stem cell transplant in October.

“I didn’t have any big hiccups, no big infections or anything like that. All was well,” she said, describing her 16 day-stint in hospital at Geelong for the transplant.

“Then I rallied around to have a luncheon for my 70th.”

That was in November, and her condition then was “only just strong enough” and she wasn’t eating much.

“I slept a lot,” she said. “I slept on the terrace in the sun, on the couch, in the caravan. I went from place to place until I got stronger. It was tiring, but it was fine. You just do what you have to do.”

Bruce and Shirley Irwin on the beach
Bruce and Shirley Irwin

Her second stem cell transplant

Shirley “took it as it came” until March (2019), when she had her second transplant. By then, her paraprotein level, which the first transplant had brought down to two, had risen to four.

“When I was first diagnosed, it was 75, which was ridiculous,” she said.

Due to her myeloma being Stage 3, she knew she was to have another ‘piggyback’ transplant.

“They had harvested enough stem cells to give me two transplants and it’s not uncommon to have two,” said Shirley.

“By the time I was due to have it, I’d forgotten the first one… but it didn’t take long to remember!

“I think I did the transplant very bravely and I rested for another couple of months after that.”

Shirley relapsed after two transplants

“Then in June, I relapsed,” said Shirley.

The myeloma had come out through the soft tissue in her left buttock.

“I thought… that’s a muscle or something… and took myself to the physiotherapist who manipulated it. Little did I realise that was the relapse.”

Shirley had expected to go through a course of treatments, starting with thalidomide, then lenalidomide, “then the next one, and the next one, and the next one… until where I am now”, she explained.

But her haematologist said, “thalidomide wouldn’t do anything, we’ll jump straight into lenalidomide with carfilzomib, but you can’t have both on the PBS”.

Shirley, Bruce and their grandsons
Bruce and Shirley with their three grandsons, Rueben, Finn and Darcy

The decision to pay for therapy

Therefore, to have both treatments together, as a combination therapy, Shirley would have to pay for one of them.

“The lenalidomide was the cheaper of the two,” she said.

“My daughter, who was with me at the time, said, ‘well, if that’s the option, that’s what’ we’ll do’.”

But Shirley wasn’t comfortable with this decision initially.

“It meant spending our money for retirement. There wouldn’t be any cash left and there would be less when we’re gone.

“It caused me a lot of stress and took me a while to come to terms with spending the kids’ inheritance on this medication, but the family assured me, ‘we don’t want anything, … we want you’.”

Bruce had worked out that the cost of Shirley’s treatment to that point, accessed via the public medical system [the Irwins don’t have private health cover or receive a pension], was around $450,000. He convinced her and she subsequently agreed, that paying $60,000 so she could have two treatments at the same time, which her specialist said, “was the best recipe for her”, was a “pretty fair go”.

Shirley got access to the more expensive treatment, carfilzomib, through the PBS, and began the lenalidomide/carfilzomib regimen in June.

“I settled into that really well,” she said.

By October, Shirley had gathered strength. She and Bruce went on a “special family holiday” to Noosa in Queensland, along with her sister, Heather, and husband, John, daughter, Paige and her husband, Dan, and their three boys, and son, Saul.

Both Paige and Saul, live close-by to their parents.

“One’s just across the bridge at Barwon Heads and one’s round the corner,” said Shirley.

Another relapse and more treatment

For nine months, Shirley did “really well” on the combination therapy until the myeloma appeared again, in April 2020, this time in her thigh bone, causing a fracture.

“I’d relapsed again,” said Shirley.

She was told the myeloma had cloned and was no longer responding to the lenalidomide/carfilzomib. She needed a different treatment, but first she had to have more radiotherapy, and before she could have that, she needed surgery to put a pin in her right leg, from her hip to her knee, to secure the bone.

That happened within days, “which was super”, said Shirley as she couldn’t weight-bear and “it was so painful”.

“It took six weeks to get over the surgery, but then after a week or so I could have the radiotherapy. Now I’m fine in that leg.

Shirley teaching her grandson, Darcy, to sew
Shirley teaching her grandson, Darcy, to sew

The protocol to access daratumumab

“Then they had to start me on something else. Pomalidomide was next on the list, so I had that for a month. Even though they felt it wouldn’t do anything, you have to go through it to get the next, daratumumab.

“That’s the only time I felt anger, when the carfilzomib stopped working. It made me angry that I had to wait before I could go on the daratumumab,” said Shirley.

Scans showed spots on Shirley’s ribs and one at the top of her arm, so she had two rounds of chemotherapy to treat these, along with six cycles of daratumumab.

“I’m absolutely fine on the daratumumab,” said Shirley.

It’s a four-hour infusion every Thursday in the day ward, and she hasn’t had any of the side-effects she experienced on previous treatments – constipation, diarrhoea, tummy or bowel pain.

“On the first day my body races a bit and I get a bit tired but that’s all and of course, I’ve lost my hair again from the chemo.

“But I think I’m at the end of the line of what I can have,” said Shirley matter-of-factly about what treatment she may have next.

“I’m just going along. I feel well and I’m happy being at home with my husband.

“You know with COVID, you can’t do anything anyway, I still don’t feel I’m denied anything. This time that I have can’t be a high time because of COVID.

“I don’t walk so much anymore, well, not a long way, I haven’t got that strength, but we’ve got a light wheelchair, which I loathe, and I have been in it to go along the river.

“And we’re near the beach and it’s quite lovely and my husband likes to walk.”

Shirley loves to quilt and has sewn since her childhood. She and her sister, Heather, ran a dedicated quilt shop together for many years at Geelong, then Shirley worked at a haberdashery at Ocean Grove prior to retiring.

“I’ve got plenty here [at home] to sew. I love all my fabrics and there is so much that I want to do. I have a sewing workroom but find it very hard to go in there,” said Shirley.

After her myeloma diagnosis, Shirley couldn’t see the point, knowing the treatment “was just to keep you alive and as well as could be”.

“I did try to ignore the term, but it really has been palliative care,” she said.

“When I was diagnosed, the diagnosis was terminal. I know there’s no cure for myeloma, but I wasn’t expected to get 15 years out of it.”

After Shirley’s first relapse, she pushed for a prognosis and was told, “well, without treatment, maybe six months”.

“But it didn’t worry me, it was just a fact,” said Shirley, who is now heading towards three years down the track.

Shirley Irwin with her extended family
Shirley and Bruce with their extended family, from left, Reuben, Paige, Dan, Finn, Saul and Darcy

Dealing with a loss of choice in her life

“So that’s why if I went into my sewing room, it was sad,” she said, describing a sadness that was “different… not scary… not complaining… just not good”.

“I didn’t like that my choice had been taken away and it’s hard knowing that.

“At first, I thought, ‘oh, what’s the point of looking out the window? What’s the point of pulling out that weed? What’s the point of… ?

“But it’s okay. I go along with it now happily, and what will be, will be.”

“I’ve brought things out of the sewing room and have done a bit of handwork downstairs,” said Shirley.

And she’s teaching her three grandsons to sew.

“When they were little, they would sit on my knee while I sewed.

“I’ve got my eight-year-old [Finn] and six-year-old [Darcy] on the sewing machine. It’s just delightful, and they are so precise,” said Shirley about her daughter’s children.

“I put a box under the foot press for Darcy, so he can reach. His chin’s just above the machine.

“I show them where to sew and where to line it up, and they love it because they’ve got strips of fabric that become something.”

Their younger brother, Reuben, aged three, watches on.

They call her Grandma and sometimes, when Shirley is well enough, all three stay the night.

“It’s lovely. I follow them to their bedrooms and kiss them goodnight and the oldest one loves to cook. When he comes to stay, he cooks my egg and asks, “how would you like your egg this morning Grandma?”.

What will be, will be

“My thing has always been, what will be will be, so let go of the fear because someone is looking after you.

“The doctors are wonderful, the treatment you’re getting is great, and it changes if you change. You only have to let them know if you’ve got a side-effect and they can make an adjustment.

“I feel for my husband in the future, but he’s really strong and is busy doing things.

“We don’t worry about the ‘when’ or the ‘what after’.”

“We have a property down at Wye River. He was a builder and he’s nicely preoccupied with plans for that. If we do sell it, it needs to be renovated and extended, so that’s what he’s doing.”

“And he’s learning to cook different dishes.”

Shirley and Bruce with their daughter, Paige, and son, Saul
Shirley and Bruce with their daughter, Paige, and son, Saul

Support from the Leukaemia Foundation

When first diagnosed, Shirley met one of the Leukaemia Foundation’s blood cancer support coordinators, Linda Saunders, whom she saw a few times and more recently, during lockdown, they have spoken on the phone.

“She was very good. I was very down at one stage and didn’t realise it when she happened to come by in the day ward,” said Shirley.

“I said I was really quite flat, and she said, ‘that won’t do, you don’t have to be’. I went to a psychologist who changed the antidepressant I was on.”

Shirley also had joined the myeloma support group for coffee and a talk – “they’d have a speaker sometimes” – and a Christmas lunch.

“Honestly that was really lovely, and I was surprised how nice it was to meet other people with myeloma, because I was thinking, I don’t need that.”

“I remember, one of the ladies said to me after my transplant, ‘oh, I was really worried about you, and you look so good’. That was just nice and that’s somebody you don’t know.”

*Bruce Irwin is a 10-year survivor of an aggressive form of non-Hodgkin lymphoma.

Research aims to improve QOL by decreasing treatment toxicity

Research aims to improve QOL by decreasing treatment toxicity

Research aims to improve QOL by decreasing treatment toxicity 

Kate van Dyke
Lead investigator, Dr Kate Vandyke is motivated by people living with myeloma whose quality of life could be improved significantly by her research work

Exciting research is examining whether a new inhibitor can be used to prepare myeloma patients for bortezomib and other myeloma therapies, to increase the efficacy and potentially decrease toxicity of these therapies.

Lead investigator, Dr Kate Vandyke is a Research Fellow in Professor Andrew Zannettino’s Myeloma Research Laboratory at the South Australian Health and Medical Research Institute, Cancer Theme.

“We’re a pretty big lab of 25 and we mainly work on factors that are associated with poor prognosis in myeloma and working out ways of targeting these factors,” said Dr Vandyke.

“We also investigate the interaction between the cancer cells and the microenvironment and pre-malignant myeloma conditions like monoclonal gammopathy of undetermined significance (MGUS) that can convert to myeloma.”

Dr Vandyke was awarded a Priority-driven Collaborative Cancer Research Scheme grant in 2019 to build on her finding that the novel N-cadherin inhibitor, LCRF-0006, can dramatically increase the antitumour efficacy of low dose bortezomib (Velcade®) treatment. This grant was co-funded by the Leukaemia Foundation, Cure Cancer Australia, and Cancer Australia.

“The project has been a long time in the making,” said Dr Vandyke.

“We’ve been working on a N-cadherin inhibitor (LCRF-0006) as a potential target for myeloma for almost 10 years.

Myeloma lab
Professor Andrew Zannettino, front right, with his Myeloma Research Laboratory staff, including Dr Vandyke, at the South Australian Health and Medical Research Institute, Cancer Theme

In this project, Dr Vandyke is looking at one particular drug (bortezomib) that’s used in the majority of myeloma patients and which causes peripheral neuropathy in about a third of these patients.

“This is a really horrible side-effect affecting the nerves in the extremities. It causes pain in the hands and feet, pins and needles, numbness, and burning sensations – it’s really nasty,” she said.

“Bortezomib, as well as targeting the myeloma tumour cells, also targets some of the long nerve fibres that go into the hands and feet. It targets the long axons of these nerve fibres and causes damage.”

Dr Vandyke’s project will look to increase the way the drug is delivered intravenously from the bloodstream to the cancer cells.

“The way bortezomib moves out of the blood and into the cancer cell is limited by the blood vessels as they’ve got a strong boundary around them that stops the blood from seeping out,” said Dr Vandyke.

“We think that this N-cadherin inhibitor is just opening up that barrier a little bit to allow the drug to get through more efficiently. It’s working more on blood vessels in cancer rather than normal blood vessels.

“You’re getting more of the drug into the cancer and can decrease the amount that’s going elsewhere in the body which should decrease those side-effects.”

Kate Vandyke in the lab
Dr Kate Vandyke: “We’ve been working on a N-cadherin inhibitor as a potential target for myeloma for almost 10 years”

The research team has just published a paper in the medical journal, FASEB BioAdvances, using a pre-clinical model showing that using LCRF-0006 effectively increases the effect of the bortezomib.

“This means we can actually use a lower dose of bortezomib and therefore we hope side-effects will stop because you’ll get a better anti-cancer effect with a lower-dose drug,” said Dr Vandyke.

The next steps will be to see if the side-effects are actually decreased and to optimise the drug to be ready for human clinical trials.

“Because this drug hasn’t been used in patients before, it would have to go through Phase I trials and safety studies before going on to larger clinical trials,” said Dr Vandyke.

“Also, because we don’t have a pharmaceutical company that’s making the drug, the next big hurdle would be funding – always a challenge.”

Dr Vandyke is motivated by the people who are living with myeloma and whose quality of life could be improved significantly by her work.

“As a scientist, you can get bogged down in the finer details of what you’re doing and you forget the big picture,” she said.

“We have a lot of engagement with patient groups that come for tours and education sessions at the lab.

“While advancements in myeloma therapies have improved survival rates significantly, toxic side-effects and treatment-related quality of life (QOL) have become increasingly important factors for patients.

“Many patients won’t want to take the drug if it’s going to have such a detrimental effect on their quality of life, regardless of the long-term survival outcome.”

Dr Vandyke is also passionate about fostering the next generation of researchers.

“I really enjoy the teaching and supervision side of things. I’ve got quite a few PhD students that I co-supervise with Prof. Zannettino and I’m mentoring a couple of junior researchers as well,” she said.

“One of them, Dr Krzysztof Mrozik, has been a real driver for this project – we couldn’t have done it without him.

“It’s also great for them to be able to talk to the patients and their carers and families and see this is why we’re doing what we’re doing.”

Dr Vandyke said she was, “incredibly grateful to Leukaemia Foundation supporters for enabling me, and my team, to do the work we do”.

“They have made this project possible.”

Dr Vandyke has used this funding support to expand her research group; to hire a technical support staff member, and support the research of a junior researcher.

“These grants are given to some of the very best emerging talent from around Australia, and I am proud to be included in this group.”

In just 10 short years, blood cancer diagnoses have risen 38 per cent and regional patients face biggest barriers to survival

In just 10 short years, blood cancer diagnoses have risen 38 per cent and regional patients face biggest barriers to survival

Tuesday September 12020 

September is Blood Cancer Awareness Month

The Leukaemia Foundation is today shining spotlight on blood cancer in Australia as it welcomes the tenth Blood Cancer Awareness Month and sharpens its focus on turning the tables on one of the country’s most common and deadly cancers. 

The number of Australians diagnosed with a blood cancer has jumped 38% in the past decade alone. Today, more than 17,300 people are expected to be diagnosed with the disease this year and sadly, more than 5600 Australians will lose their lifean increase of 27%¹  

Projections show that while 110,000 Australians are living with blood cancer today, that number will more than double to 275,000 by 2035. Blood cancer is also expected to claim the lives of 186,000 Australians in this time². 

The Leukaemia Foundation’s Acting CEO Alex Struthers said that, sadly, survival could also depend on where you live. Last year’s first-of-its-kind State of the Nation: Blood Cancer in Australia report revealed a 13 per cent gap in survival rates between patients in regional and metropolitan areas (5%), and between states and territories (8%).  

“The State of the Nation report showed us that regional blood cancer patients aren’t receiving the crucial diagnostics and specialist care they need, when they need it and are more likely to face barriers in getting this care,” she said. 

Over half of all blood cancer patients living in regional and remote areas are more likely to wait over a month to see a haematologist after presenting to a GP, and a third are unsure about their treatment plan. One in four of these patients also don’t know where to go if they have more questions about their blood cancer. 

“By breaking down these barriers and removing variations in access to best practice treatment and care, Australia could minimise mortality and potentially save up to 22,000 lives by 2035. 

Ending the postcode lottery faced by regional patients and turning the tables on blood cancer in this country is a priority for the Leukaemia Foundation.” 

The Leukaemia Foundation’s release of the State of the Nation report last year led to Federal Minister for Health, The Hon. Greg Hunt MP establishing the Blood Cancer Taskforce – a unique collaboration of some of the nation’s top blood cancer experts and leaders to transform care and support and break down the barriers to a cure for all Australians living with blood cancer. 

The Taskforce is now preparing to launch the National Strategic Action Plan for Blood Cancer – a cuttingedge national blueprint to save the lives of Australian’s facing blood cancer with a bold vision of zero lives lost to the disease by 2035, due for release soon. 

Ms Struthers said the unique collaboration of the Blood Cancer Taskforce anthe development of the National Strategic Action Plan for Blood Cancer represented a real opportunity to change the face of treatment, care and ultimately survival for Australians facing a blood cancer diagnosis.    

“Australia has strong foundations in regards to access to new therapies and universal access to a national health care system, but with a significant increase in blood cancer incidence – even during a global health pandemic – a clear, evidence-based plan is now required to tackle the human and financial toll of blood cancer in our country,” she said.  

There is no onesizefitsall solution. We have excellent health systems and services across Australia which are achieving remarkable results in improving survival rates and treatment for people living with blood cancer, however we need to work together if we are going to meet the challenges we face and help all Australians with blood cancer not only survive, but to live the best quality of life possible. 

With less than 50% of people living with blood cancer given a written care plan, and only 1 in 5 people having access to a clinical trial³, there is work to be done to improve supportive care and access to treatment for Australians living with blood cancer. It is our hope that the National Action Plan, once implemented, will pave the path to saving lives as well as bridging these gaps. 

The Blood Cancer Taskforce has delivered the National Action Plan to the Federal Government and will continue to work alongside the government to move towards the launch of the plan soon. 

Leukaemia Foundation spokespeople are available for comment by contacting the Leukaemia Foundation media team on media@leukaemia.org.auImagery is available here or through the same contact.  

About Blood Cancer in Australia 

Incidence and Mortality4: 

  • Every year, 17, 321 Australians will be newly diagnosed with blood cancer such as leukaemia, lymphoma and myeloma. This is equivalent to 47 people every day or one person every 31 minutes. 
  • Blood cancer does not discriminate. It can develop in anyone, can occur at any age and at any stage of life across all states and territories, from children to adolescents and young adults to working adults with families and older Australians.  
  • When combined, blood cancers are among the most frequently diagnosed cancers in Australia, and the most significant cause of non-preventable cancer death.  
  • One in 10 Australians diagnosed with cancer will have a blood cancer. 
  • Over 5,600 people in Australia are expected to lose their life to blood cancer or related blood disorders this year. This is equivalent to 15 people per day in Australia. 
  • Blood cancer is one of the highest causes of cancer death in Australia, claiming more lives than breast cancer (3,031ii) and melanoma (1,375) combined.  
  • Approximately one in nine cancer deaths in Australia will be due to blood cancer. 
  • There are no screening programs available for blood cancers, and there is no way to prevent blood cancer through lifestyle change. 


  • Based on AIHW data, the State of the Nation report identified issues with the undernotification of blood cancers in Australia and anticipates that more than 110,000 people are currently living with blood cancer or a related blood disorder in Australia today.  

Childhood Cancer:  

  • Blood cancer (in specific leukaemia and lymphoma), remains the most commonly diagnosed childhood cancer (aged 0-14 years) in Australia, accounting for over 40% of all diagnoses.  

About the Leukaemia Foundation 

The Leukaemia Foundation stands with Australia to help cure and conquer blood cancer – with care. Together we are attacking every blood cancer, from every direction, in every way we can. We stand beside every Australian to be their voice and their someone-to-turn-to, fighting to get them access to the best care. We also accelerate research that’s delivering rapid advancements in blood cancer diagnosis and treatments. Plus, we provide services and support that empower people living with any blood cancer to live well after diagnosis. You can learn more about the Leukaemia Foundation and blood cancer at leukaemia.org.au   

– ENDS –

Subscribe to receive the Leukaemia Foundation’s news and updates here.

Remembering Jacob: ‘Leukaemia Sux!’

Remembering Jacob: ‘Leukaemia Sux!’

The McLeod-Steinmetz family
The McLeod-Steinmetz family photographed for a newspaper article in 2014, from left, Jon, Jonathon, Jacinta, Jacob, Deanne and Ryan

Since losing their son, Jacob, to leukaemia 15 years ago, Deanne and Jon McLeod-Steinmetz and family have honoured his memory with special rituals of remembrance, including the Leukaemia Foundation’s Light the Night.

Jacob was 12 years old when he was diagnosed with acute myeloid leukaemia (AML) in November 2003, and his mum, Deanne, recalls the feeling of shock. He had just played at a representative soccer tournament, and throughout his life, had rarely been sick.

Jacob McLeod-Steinmetz
Jacob with the family dog, Bejay, in 2014

“He was my healthiest of four children, always a veggie-eater, and had never had a cold or flu his entire life,” remembers Deanne, of the outer Brisbane suburb, Alexandra Hills.

After four cycles of chemotherapy, Jacob was transferred to the Royal Brisbane Hospital for more intensive treatment, but ongoing roadworks meant Deanne always had to call on the way, saying they were stuck in traffic.

“That’s when the Leukaemia Foundation contacted us and offered us our own unit at their Herston Patient Accommodation Village,” she said.

Jacob McLeod-Steinmetz on the soccer field before his diagnosis
Jacob McLeod-Steinmetz on the soccer field before his diagnosis

“It was just down the road and that was a godsend as the doctors said we needed to be close to the hospital, considering the terrible reactions Jacob kept having to the chemo.”

Due to Jacob’s poor response to treatment, it became apparent that he needed a bone marrow transplant.

“No one in our family was a match,” said Deanne, but a Melbourne woman was found who could be his donor.

Deanne sent her a thank you letter and a little angel necklace.

“Every year on the date of his transplant, I put a message on Facebook saying we are forever grateful to the mother of six that helped us.

“She gave us four more months with our boy.”  

In February 2005, 92 days after his transplant, Jacob relapsed, and his parents were told “to go home and make arrangements”.

“We gave Jacob one last weekend before telling him the news. We all went on a family trip to Sea World and had a great time,” said Deanne.

“On the Monday, we broke the news to him, and he asked us to bring all the other kids into the room.

“Jacob’s the youngest and he sat them all down, put his arms around Jacinta (then 19) and Ryan (15), looked at Jonathon (18) and said, ’my leukemia’s back and I’m going to die. But it’s okay, we’re not going to stop fighting’.”

In the following months, the family dedicated most of their time to making memories with Jacob.

“We went on our Make a Wish trip to New Zealand so Jacob could see snow and our daughter’s boss paid for a trip to Disneyland,” said Deanne.

“We had some amazing moments during that time and made memories that we will treasure forever.”  

By mid-2005, Jacob’s condition was critical. His leukaemia was becoming too much for his body to handle.

“We were able to get him home, far away from the hospital, and he left us peacefully at 5:08pm on 16 June 2005, just a day short of his 14th birthday,” said Deanne.

Since Jacob’s passing, Deanne and her family have established special ways to honour his memory.

“On the first anniversary we went to Jacob’s three favourite soccer fields in Brisbane to spread his ashes as he had wished,” said Deanne.

“It was such a Jacob-type of day with gorgeous weather and all these funny things happened that were very reminiscent of the way he was, like balloons going crazy on the field.

“Now every year on 16 June we go to what we call ‘Jacob’s Beach’ at Wellington Point where we have fish and chips and watch the sun set at 5:08pm.

“It’s really important for us to mark and acknowledge that moment when our world changed forever.” 

Jacob’s brother, Ryan, in his ‘Leukaemia Sux’ shirt at the inaugural Light the Night in 2008
Jacob’s brother, Ryan, in his ‘Leukaemia Sux’ shirt at the inaugural Light the Night in 2008

“I go again at 6am the next day to watch the sun rise on his birthday and we always make cake and pasta for his birthday tea,” said Deanne.

The whole family has attended the Light the Night event nearly every year since it started in 2008 and they made special ‘Jacob: Leukaemia Sux!’ shirts to wear.

“Jacob used to wear shirts with ‘Leukaemia Sux!’ on them and we thought we would continue the tradition,” said Deanne.

“It can be a bittersweet night, but I love going to meet up with all the mums of the survivors who were Jacob’s age.

“We all have a cuddle and chat about how their now-adult kids are going.

“I’ll often also approach someone who you can tell is a newbie to the event and is holding a gold lantern.

“I’ll ask them about who they’re there for, and tell them a bit about my story. I think they really appreciate the chance to talk openly about the person they’ve lost,” she said.

“It’s good when members of the public stop you on the walk and ask what all the lanterns mean.

“I’m always proud to explain we’re supporting the Leukaemia Foundation and research into the disease that took my son’s life too early.”

Deanne, who was unable to attend Light the Night last year, was excited to hear the event would be virtual in 2020.

“This will be a fantastic way to make sure everyone is included, no matter where you are or what situation you’re in,” said Deanne.

“Many families going through blood cancer know how risky it is, just in normal times, not only in the middle of the pandemic, for patients to be in a crowd or public place.

“You feel like the crazy, paranoid mum getting everyone to sanitise constantly, but it really is a matter of life or death for your child.”

Deanne and Jon have also attended the Leukaemia Foundation Bereaved Parents’ Weekend in the past where they formed strong bonds with others who have lost a child.

“Those weekends were amazing and I’m still in contact with a couple of the mums we met,” she said.

“What I enjoyed was not having to put on a front that you’re fine because everyone else knows exactly how you’re feeling.

“You can be angry, sad, crying, or laughing hysterically and there’s no judgment whatsoever.

“You can’t really talk about it too much with others in your life because their eyes glaze over, or they get embarrassed and change the subject. I find that especially when people find out it’s a child you’ve lost.

“Both my parents have passed away and people still easily say to me, ’I’m so sorry about your dad’.

“But after Jacob passed, when I would see some of his mates’ mums in the mall, they would literally turn around and run the other way or duck into a shop.

McLeod-Steinmetz siblings at Wellington Point
The McLeod-Steinmetz siblings at ‘Jacob’s Beach’ at Wellington Point, in 2011, from left, Jonathan, Jacinta and Ryan with a photo of Jacob

“That actually really hurt, and my advice would be that it is much better to say the wrong thing, then nothing at all.” 

Deanne still misses Jacob every day and is dedicated to making sure he is never forgotten, which she considers “the greatest fear for every parent who has lost a child”.

“Jacob will always be my ‘almost’ 14-year-old, and not a day goes by that I don’t think about my cheeky and mischievous boy up there watching over us,” said Deanne.

Music therapy – creative support for the bereaved 

Music therapy – creative support for the bereaved 

Angela Delaney
Music therapist, Angela Delaney

Angela Delaney is a music therapist who is privileged to work with people to explore music-facilitated experiences in deep and meaningful ways where words often fail. In this article, Angela provides insight into how music therapy can contribute to the grief process.

Grief and loss

The death of a loved one is heartfelt like no other, an experience that goes beyond words.

Grief is the natural, normal human response to loss. It affects every part of us – our body, mind, spirit, relationships and feelings, yet can leave us feeling anything but normal.

Understanding of grief and bereavement has shifted. For many, the human process of understanding and living with loss is never really final, complete or resolved, effecting each person in unique ways.

People grieving a death often feel that no one understands what they are going through. We have learnt from those who are living through grief and loss that the help they appreciate most comes from sharing with others who also are grieving a death.

Creative approaches, particularly group music therapy, are documented to effectively support bereaved individuals.

Angela Delaney
Angela Delaney: “many therapists use music with grieving individuals of all ages”

What is music therapy?

Music therapy is an allied health research-based profession that is practiced worldwide. Music therapists apply creative and professionally informed music to support the health, functioning and wellbeing of people of any age [1].

Why use music in in this context? Music is a human phenomenon; an innate human ability, and also the only sensory experience that can activate all areas of the brain simultaneously [2].

Why music?

Music plays a role in the lives of many people. Music helps us to express feelings and memories and to connect with others, which is an essential part of the grieving process.

The elements of music rhythm, melody, harmony, dynamics, timbre, and form address our basic sensory needs. Amidst the power and unpredictability of grief, music and music therapy have the capacity to provide predictability and comfort; ameliorate distress; and provide adaptive coping and wellbeing [3-5].

Music therapy also provides opportunity for choice and control. It enables bonding, facilitates stimulation and relaxation, enhances communication, and fosters positive experiences [6-8]. Using music therapeutically with peer group support can support participants to discover their own way through grief.

How does music therapy work?

To understand the relevance between music and the process of grieving, it is important to be aware of the effect of music on the brain, and the relationship between music and the expression of emotions.

The neurological pathway for sound allows music to affect those structures in the human brain most responsible for emotional behaviour; the hypothalamus and limbic system.

This neurological process may explain why couples decide to choose a song as ‘their song’ to remind them of the emotion of love for each other. It also may explain the effect that movies can have on an audience, through the use of music to elicit sadness, fear, or joy.

Knowledge of the effect music has on the brain has led many therapists to use music with grieving individuals of all ages. The use of original therapist/group-composed songs is effective in decreasing the physical grief symptoms during bereavement and to elicit suppressed emotions due to grief. Research has shown us that specific music therapy interventions such as songwriting and improvisation, support positive growth and the processing of grief.

My role as a music therapist involves using music and all its elements, along with verbal processing to:

  • engage and support the expression of grief and bereavement through group improvisation and songwriting;
  •  facilitate spiritual connection by experiencing pleasure, ‘normalcy’, creativity, and community;
  • create recordings and songs together; and
  • facilitate connection with others and reduce isolation.

Music offers expanded opportunities for aesthetic experience and meaningful communication through its capacity to activate more areas of preserved neural function.

For 10 years, I have had the honour of walking alongside bereaved parents, using music therapy in groups to provide a safe and comfortable environment with others who have also experienced the death of a loved one.

Music therapy in a group session

Following drumming improvisation, the group explores various experiences and emotions around their lived experiences of grief, often allowing emotions to surface that may have been suppressed. Many participants report improved emotional health following a music therapy session.

The depth of personal expression shared amongst the group reminds me of the depth of resilience and bravery we may not even be aware we possess, as I witness and support their exploration of grief.

The level and intensity of emotions expressed during the session varies and at times can sound and feel chaotic, apprehensive, and explosive. Using music involves each group member directly, increasing emotional awareness, stretching imagination, and deepening affective resonance. Songwriting then allows the lives of the bereaved to be told, and acknowledges the existence and the individual nature of grief.

Music therapy has potential to contribute significantly to the experience of grief and bereavement. Music therapy is a pivotal ingredient, to bring comfort, resolution and spirituality and to provide a compassionate space for a family’s transition to the changing interpersonal connection after death.

Song lyrics from a bereaved music therapy group.

The sun rises, the sun sets
Your wings envelope me
I feel a sense of peace
Everything is ok.

Tears of joy
Tears of sadness
Make our own reality.

Feeling so hollow
Like a deep emptiness
So many regrets
As the world moves on by.

The following piece of beautiful music, Elegy for the Arctic, is from Ludovico Einaudi’s album, 12 Songs from Home. It is best watched, at least for the first listen.

Ludovico Einaudi performs Elegy for the Arctic in 2016 on a floating platform with a glacier backdrop in Svalbard (Norway). Watch on YouTube here.

And during an Australian tour in 2020 here.

Listen on Spotify here.


  1. Australian Music Therapy Association. What is Music Therapy [Internet]. 2012
  2. Sacks OW. Musicophilia. 1st ed. New York: Knopf; 2007.
  3. The contribution to palliative care of allied health professions. In: Watson MS, Ward S, Vallath N, Wells J, Campbell R, editors. Oxford Handbook of Palliative Care. 3rd ed. Oxford: Oxford University Press; 2019.
  4. Daveson BA, Kennelly J. Music Therapy in Palliative Care for Hospitalized Children and Adolescents. J Palliat Care. 2000 Spring;16(1):35-8.
  5. Hilliard RE. Music therapy in paediatric palliative care: complementing the interdisciplinary approach. J Palliat Care. 2003 Summer;19(2):127-32.
  6. Amadoru S, McFerran K. The role of music therapy in children’s hospices. Eur J Palliat Care. 2007 14(3): 124-127.
  7. Duda L. Integrating music therapy into paediatric palliative care. Progress in Palliative Care. 2013 12(2), 65-77.
  8. Lindenfelser KJ, Hense C, McFerran, K. Music therapy in paediatric palliative care: family-centred care to enhance quality of life. Am J Hosp Palliat Care. 2012 May;29(3):219-26. doi: 10.1177/1049909111429327. Epub 2011 Dec 4.

Meet our national Grief and Bereavement Services team

Meet our national Grief and Bereavement Services team

To help loved ones through perhaps the most challenging experience of loss in their lives, the Leukaemia Foundation has grief and bereavement support staff across Australia.

They can help you understand your grief, talk through your feelings, connect with a support group, or identify when an expert may be able to help. Each of our staff members has a background in nursing or allied health and is experienced in offering support.

To access grief and bereavement support, call 1800 620 420 or email us at grief@leukaemia.org.au

Shirley Cunningham
Shirley Cunningham

Shirley Cunningham, Queensland

I am the national grief lead for the Leukaemia Foundation, a role I share with Donita Menon. I have worked for the organisation for more than 16 years, starting as a blood cancer support coordinator in June 2004. I have extensive experience as a registered nurse and have dedicated most of my career to caring for adults and children diagnosed with solid tumours and haematological diseases.

I have always been drawn to the psychology of people and those dealing with loss, grief and bereavement, and became the grief manager for Queensland 18 months after I started at the Leukaemia Foundation.

In my current role, I work with families in Queensland and those referred nationally for counselling. It is an honour to counsel and support our clients and my ongoing passion is caring for individuals and their families diagnosed as well as those bereaved by blood cancer.

I have studied extensively and continue to increase my knowledge in these areas. I have a degree in nursing, graduate certificate in paediatric nursing, master’s in health loss and grief and a master’s in counselling and Level 1, Gottman Institute training for couple therapy.

It is such a privilege to work for the Leukaemia Foundation and I am humbled to be invited, even for a small time, into the lives of patients and families at such a devastating time. Now with the organisation having grief support available nationally, more families are being supported in their grief than at any other time in our history.


Donita Menon
Donita Menon

Donita Menon, New South Wales and Australian Capital Territory

I have been at the Leukaemia Foundation, working as a grief and bereavement counsellor since 2009. In my role as the national grief lead, alongside Shirley Cunningham, I work with clients who have a blood cancer diagnosis, members of their family, and those who are bereaved. I also have worked for Calvary Health Care as a pastoral care worker, in palliative care and in rehab. And prior to that as a primary school teacher for 26 years.

The challenges that individuals and families go through with a diagnosis of a blood cancer is unimaginable. It can be a very lonely space at so many different levels. To navigate life without the person you love can feel impossible at times.

My hope in the work I do, is to provide a safe space for every client to share and process their pain and to work hard to understand each client’s world through their eyes. I believe that every individual has the answers within themselves and through counselling can find a space to grieve, feel the pain and gently unlock a pathway to navigate their life. Going gently, being kind to oneself, nurturing and nourishing oneself and self- compassion are just some of the pathways to healing in grief.

Jane Anderson
Jane Anderson

Jane Anderson, Victoria and Tasmania

I’m currently the Leukaemia Foundation’s Blood Cancer Support Coordinator in southern Tasmania and have recently become the Grief and Bereavement Lead for the states of Victoria and Tasmania.

I completed a Bachelor of Nursing at the Royal Hobart Hospital in 1986. I then went on to complete a Graduate Diploma in Cancer Care Nursing in 1996 and have more than 35 years professional experience in haematology, palliative care, and bone marrow transplant nursing.

I have worked in a variety of haematology oncology specialty areas including The Royal Marsden in London, and as the Bone Marrow Transplant Coordinator for Tasmania at the Royal Hobart Hospital.

I joined the Leukaemia Foundation in 2004 to establish an office in Tasmania and focus on improving support services for patients and families affected by blood cancer.

Passionate about the importance of holistic care for patients and their families, I have always advocated that Leukaemia Foundation support continues for families when a loved one with blood cancer dies. My new lead grief role enables me to offer grief and bereavement support to loved ones.

Shelley McLean
Shelly McLean

Shelly McClean, South Australia and Northern Territory

I have a Bachelor of Midwifery, a Master’s in Social Work and have been at the Leukaemia Foundation for 18 months.

I remember during my midwifery training and was working with a midwife to care for a mum who had previously had a still birth. The midwife was insensitive to the mother’s grief and fear, which retraumatised the young mum and made her birth distressing.

On another occasion, I worked with a midwife who had such skill and compassion, that she helped the family welcome their new baby in safety, while honouring their child that had died. That was when I knew that it was sacred work, and I would be honoured to share in those moments of grief and heartache, but also in the joy of reminiscing and cherishing moments and memories.

That day, I made a commitment to be the kind of person that would stay and allow the tough conversations, and the messiness of grief and loss because deep sorrow requires deep compassion.

I am also involved in the South Australian Women’s Grief Group and wanted to extend this healing on a national level. Being beside someone, allowing them to tell their story and helping to develop their own new meanings for their lives, is very rewarding.


Jacqui Baverstock
Jacqui Baverstock

Jacqui Baverstock, Western Australia

I did my Registered Nurse trainings (RGN/RSCN) in London where I then worked in the community and then as a part of a children’s oncology and haematology team.

I started working at the Leukaemia Foundation in December 2016 and was delighted to recently become a part of the grief and bereavement team. I have always had a big interest in grief and bereavement through my close work with the families of patients.

Jenni Bourke
Jenni Bourke

Jenni Bourke, Victoria 

I am a blood cancer support coordinator with the Leukaemia Foundation, based in Victoria, and recently became the grief and bereavement lead in Victoria. 

During my time at Leukaemia Foundation I have had some extraordinary conversations with the people who use our service every day. Many of these reflect themes of adjusting to constant change, managing the unknown, and living every day with the big and little losses that accompany a blood cancer diagnosis.  

Being on the national grief and bereavement team is an important part of providing individual support to families through what can be an intense and unpredictable time of major adjustment. I have a passion for providing holistic care and bring many years of clinical experience to this role.  

I work as an occupational therapist and developed my haematology skills during the 12 years I worked at Peter MacCallum Cancer Centre. The desire to actively provide emotional support as part of routine care has led to much learning, including further study in palliative care, and grief and bereavement I am a member of the Australian Grief and Bereavement Centre and attend many of their workshops and conferences to ensure my practice remains relevant. 

Providing a gentle and compassionate space to allow people to express their story and feelings, to explore existing strengths, to connect with the deep need to grieve and reshape their connection with their loved one, are all elements of learning to live with loss.  

“Trev is still looking after me” says Pauline

Trevor and Pauline with their dog
Pauline and Trevor with their beloved dog, Harry: “We loved him so much. He was so special to us.”

Had Pauline Vedelago been told, 3½ years ago, that she would no longer have her husband, dog, house, job, or live in Bundaberg, she would have said, “are you crazy?”.

“My life was so different 3½ years ago,” said Pauline, 58, who is retired, volunteers two days a week, and writes to her late husband every night, telling him about her day.

While there are many blood cancer success stories, unfortunately there also are stories where treatment is unsuccessful, the disease mutates, and in the end, devastatingly, there are no further options.

Sadly, this happened to Pauline’s husband of 31 years, Trevor Boyd, a teacher-librarian whose prognosis was “pretty good” when diagnosed with non-Hodgkin lymphoma (NHL) in May 2017, aged 55.

Over the next 2½ years, the couple, went back and forth from their home in Bundaberg to Brisbane, for tests and treatment.

Pauline and Trevor in hospital
Pauline with Trevor in January 2018 after his admission to hospital for his stem cell transplant

Trevor’s determination to beat blood cancer

When Trevor didn’t respond to his first or second lines of chemotherapy, he had an autologous stem cell transplant in January 2018, which his haematologist said gave him the best chance of a long-term good outcome.

After six weeks in Brisbane for the transplant, and being told that Trevor looked to be “in the clear”, they went home, but not for long, because an infection in Trevor’s Hickman line turned out to be serious.

“It was called mycobacterium fortuitum, which always made me laugh because I thought it doesn’t feel very fortunate,” said Pauline, so back they went to Brisbane for another month for round-the-clock intravenous antibiotics.

Finally, the couple returned home and glimpsed normal life again – camping together, catching up with friends, exercising regularly, and Pauline started her social work again, part-time.

“Life was starting to look good,” she said.

Trevor was in remission and he went back to work for the last six weeks of the 2018 school year.

“Trev led a very quiet life, very similar to what people do now, really, socially isolating. Any friends who were coming over, we’d make sure they were all well and kept their social distance,” said Pauline.

This year, when coronavirus started, Pauline said it felt really familiar: “This is what we used to do all the time. It felt like history repeating itself”.

Towards the end of 2018, Trevor started to feel unwell. He had fevers and fatigue and assumed it was a virus he’d picked up from the school kids, but after a month, when he didn’t get better, his GP suggested another round of scans.

Pauline and Trevor after Trevs knee surgery
The Boyds, in January 2017, after Trevor had knee surgery and just months before his diagnosis

Another relapse and two different diagnoses

On his 57th birthday, on December 29, he got the news. He had a mass on his lung and within days the Boyds were back in Brisbane. Trevor was diagnosed with a different blood cancer – chronic lymphocytic leukaemia – and started a new regimen of chemo. Soon, however, scans showed that it wasn’t working.

Miraculously, a new drug (venetoclax) had been listed on the Pharmaceutical Benefits Scheme that week, which Trevor started immediately, combined with another immunotherapy, not yet approved in Australia, and which cost the couple “several thousand dollars”.

Then, genetic studies revealed that Trevor had “a really bad mutation, c-myc, which doesn’t have a good prognosis”. An allogeneic stem cell transplant was his only option. Disappointingly, none of his three sisters was a match, and he was running out of time to find a matched unrelated donor.

Next, he was told he actually had Richter’s transformation; a disease that looks like NHL but is “something much worse” because it constantly mutates, and CAR T-cell therapy in the U.S. would give him the best chance of survival, providing he was accepted on to a clinical trial in Seattle.

“If we got the okay, we had to go right away, and have a spare half a million dollars!” explained Pauline. But first they needed passports, urgently, at $500 each.

“Mine had expired and Trev hadn’t had one for 20 years.”

They also were in the middle of selling their home in Bundaberg.

Trevor and Pauline in Adelaide
Trevor and Pauline holidaying in Adelaide

“We just thought, ‘oh well, we can do that’. We’d borrow money from everyone and anyone, get our super, and pay people back.

“That’s the kind of pressure you’re under. I thought I was going to go mad. I could barely cope,” explained Pauline.

“When we had the relapse in January 2019, I couldn’t cope… I had a real little meltdown.

“Trev was really tough, and he was stoic all the way through, but I started to fall apart.

“I got myself together, went to counselling, went to my GP, started antidepressants… because I’m a social worker; I know what you’re supposed to do to look after yourself.

“I thought, all right, I have to be like Trev, to be strong. We just have to keep going.”

Leukaemia Foundation support

Pauline had been in contact with the Leukaemia Foundation and one of our blood cancer support coordinators, Sheila Deuchars.

“Sheila was great. She put us in touch with some Australians who were over there [in the U.S.] at the time doing the same thing and we had been in contact with them.”

But, at the last minute, Trevor was told he wasn’t eligible for the American trial because his disease was mutating.

“That was so disappointing, but we were still hopeful,” said Pauline.

“Trev was writing to professors all over America to see what trials were coming up that targeted the CD20 mutation.”

Then, another miracle, a clinical trial was starting… in Brisbane! Trevor just had to have all the tests and tick all the boxes, which he did. He was eligible, and the trial paid for their travel and accommodation expenses.

“Fantastic, we could fly to Brisbane instead of driving or taking the train,” said Pauline.

Trevor started the trial in August [2019] but, after six weeks, he’d had “absolutely no response”.

“That’s when we had the big conversation with the specialist,” said Pauline.

“He said, ‘we’ve thrown everything at it… you might as well go home and make your end-of-life preparations’ blahdy blah, but there was still one option, ‘we could try high dose chemo as a last resort. Go home and think about it’.

“I knew Trev was never going to give up. He was going to keep fighting. That’s the way he had to go.

“He still was feeling pretty well, still working around the house and gardening.”

Pauline said her worst fear was that Trevor would go to 4W, the hospital ward for people on high-dose chemo, and be transferred to 4A, the palliative care ward.

“For three years, on and off, I’d look at that ward. My sister who died of cancer in 2013 had been in that ward,” she said.

When Pauline suggested they go on the Ghan, “something we’ve always wanted to do”, his response was ‘no, I can’t go on a holiday. I won’t enjoy it if I’m not fighting for my life’.

“I said to him, ‘of course, I’ll support you, whatever you decide’.”

Trevor started high dose chemo in September [2019] and had radiation as well.

“It was pretty awful, just one nightmare after the other, in October, November, until early-December when it didn’t work,” said Pauline.

“Trev was in hospital all that time. I just wanted to be with him, I didn’t want to be anywhere else.

“We were so grateful for the Leukaemia Foundation. I took up Sheila’s offer of a unit at Herston. It was so lovely. I’d get the bus in [to the hospital] in the morning, I could stay as long as I liked, and get an Uber at night to come home. It was so easy.”

It was Pauline’s 58th birthday on December 10, “we got some lovely photos”, and Trevor died eight days before his 58th birthday.

“While all that was happening, we sold the house, and the contract was a story in itself!

“Harry, our dog, died too last year, in April. We had to put him down. We loved him so much. He was so special to us because we don’t have children.”

When Pauline spoke to Living Well With Grief, six months after Trevor’s death, she said it “was wonderful” living in a granny flat that adjoined Trevor’s sister’s house in Brisbane, for the time being.

“I’ve been on my own, but not really alone. I’ve got contact with family, but without feeling like I’m in the way,” said Pauline.

“I appreciate the support and having someone who cares whether you get up in the morning, because so often I just didn’t see the point.

“But every day I’ve got out of bed, because Trev’s sister and her husband have been so lovely to me, I don’t want to worry them about anything.”

Pauline and Trevor on Paulines birthday
Pauline Vedelago celebrating her birthday with Trevor in their Leukaemia Foundation Unit at Herston, eight days before he died

Grief support there when needed

Since Trevor’s death, Pauline has been supported by Shirley Cunningham from the Leukaemia Foundation’s grief and bereavement team.

“I said to Shirley, ‘I’m the perfect client. I’ll do everything right to try and get through this. I’m not going to let it beat me’.

“But I still feel really sh*t, so that’s why I’ve come to counselling. I had my first appointment about two months after Trev died and I knew I wasn’t doing too well, and it was really lovely talking to Shirley.

“I was a social worker for 30 years. I’d never done grief counselling but thought I understood grief a bit. When my sister died, I thought, ‘okay, this is how it works, this is pretty sh*t, but okay, and when Trev was diagnosed, and then when I knew he wasn’t going to make it, I thought I was prepared.

“But you know, losing the person that you love most in the world, nothing really prepares you for that.

“One positive thing about cancer is that Trev and I had plenty of time together to say all the things you want to say, and I’m very appreciative of that, but it certainly doesn’t prepare you for the depth of grief you feel afterwards.

“Trev was really organised, making sure our super was good, so financially, I’m quite comfortable.

“We had grey nomad plans. We’d done a few three-month camping trips around Australia and we were just going to do more of the same… four-wheel driving.

“I do find that hard, letting go of the life that you thought you were going to have.

“It’s just accepting that things have to change. It’s trying to be mindful, trying to live in the present rather than focus so much on longing for the past.”

Pauline said she has experienced a loss of identity: “I was a social worker, but I’m not. I was married, but I’m not. I used to live in Bundaberg, but I’m not. I used to have a house, but I don’t.

“I used to be married, but now I’m a widow. I’m thinking, well, who am I? Who am I without Trev?

“I had a good chat to Shirley about regression, which I’d read about.

“You are stripped back to your most vulnerable self. I felt like I was in my teens again for quite some time. I felt so vulnerable. I didn’t want to talk to anyone, I didn’t want to go out.

“Then I did this complete about-turn. I became the Academy Award recoverer, where you say to everyone, ‘I’m fine. I’m going okay. Oh, not too bad’.

“I did that for a couple of months, because you want them to think you’re okay. You want them to think well of you, that you are recovering.

“I said to Shirley, ‘you’re the only person I’m crying with these days and I don’t think that’s good. I’m just covering up all the time’, so we had a good chat about that and what I should do.

“Grief is a very weird thing. I realise now why you do certain things and I’m very normal. The book I’m reading now is The Year of Magical Thinking which is about the difficulty you have with emotional acceptance.

“I was there when Trev died, but you still have so much trouble accepting it and you still think maybe somehow they’re still alive. I’d wake up in the morning thinking Trev was still alive.”

Making a new life

Pauline has decided to stay in Brisbane, plans to buy her own home there, and is putting things in place.

She has started going to church again and says, “I do have a faith, I do believe in an afterlife, and that has helped”, has become more active in her yoga practice, and has started bike riding with her brother-in-law.

She regularly sees her two brothers who live in Brisbane and has reconnected with a few friends, including an old uni friend she hadn’t seen for 30 years. And Simone, who she met when her husband Pete was sitting beside Trevor during chemo, has become a good friend.

Pauline had always wanted to “do something with people who care for wildlife” in her retirement and due to some divine intervention, she is volunteering two days a week at a koala sanctuary, which is helping her to “just be in the moment”.

And she’s started a journal.

“I’ve found journaling very helpful. At first, when I was just so miserable, I kept writing about how terrible I felt, and I didn’t know if that was helping. Then I received a random text from someone that said, ‘remember the joyful times you had with Trev’, and I thought, that’s what I need to do.

“So, every night I made sure that at the end of my journal writing, I’d write down a happy memory and thank Trev for that. Sometimes that was really hard, but I did that for months.

“And now I don’t do the happy memories anymore, I just do gratitude now. I’m still writing to Trev, but I finish with things that I’m grateful for that happened during the day,” said Pauline.

“I do believe Trev is still present in some form, in some way, and is still looking after me somehow. And I just figured, well, this is my way of communicating with him.

“I don’t think I’ll keep writing to him for the rest of my life, but I’m determined to keep going with it for this year.”

Pauline and friends kayaking Coonarr Creek, Bundaberg for Trev’s tribute paddle
Pauline and friends kayaked up Coonarr Creek, Bundaberg for Trev’s tribute paddle in March 2020

A tribute to Trev

Back in Bundaberg, Pauline used to kayak with a group of women, and one time Trevor joined them.

“One of my friends suggested it would be nice to have a paddle tribute to Trev, which I thought was a great idea.”

The tides were checked, a date was set, and Pauline went up to Bundaberg for the weekend.

“We ended up with 13 people and two dogs, and I decided to take half of Trev’s ashes for a little ceremony.

“Bundaberg ginger beer was Trev’s favourite drink before he went off all sugar, and I put half of his ashes in six Bundy ginger beer bottles, one for each of his best mates, and me, which we carried upstream to a lovely, quiet place.

“I thanked everyone, then they picked up their bottles and I put his ashes in the creek.

“I was so happy I did that and again, divine intervention, it was at the beginning of March, only a couple of weeks before coronavirus kicked in, and I thought, ‘oh, Trev, you’re looking after me’.”

Pauline wanted special mention made of how grateful she and Trevor were to the Leukaemia Foundation, “for everything they helped us with”. In particular, the practical support of “those two months when I lived in the Leukaemia Foundation unit”.

“I’m a regular donor now and I’ll be making a bequest in my will when I redo it again. And I’ve registered for Light the Night.”

Jennie’s had baby George since her MPN diagnosis

Jennie’s had baby George since her MPN diagnosis

Jennie Wigginton and her family on holiday at Wilson’s Promontory in September 2017
Jennie Wigginton and her family on holiday at Wilson’s Promontory in September 2017

Jennie and Russell Wigginton had to put their plans to have a second child on hold when Jennie was diagnosed “out of the blue” with essential thrombocythaemia (ET) in May 2015. 

Their son, Austin, was two at the time, and the Wiggintons had been trying for a second child for six months.

“We got pregnant quite quickly with our first,” said Jennie, now 39, a social worker who also runs her own business as a fitness instructor and personal trainer.

Jennie Wigginton in the park
Jennie Wigginton: “I do what I can to stay healthy, and ultimately my family is my inspiration”

In the build-up to her diagnosis, Jennie had been getting severe headaches.

“Really painful headaches, where you could take Panadol and Nurofen and it wouldn’t even touch the sides,” she explained.

Russell, concerned she may be diabetic, suggested she go to the doctor, and three days after having blood tests, Jennie got a call saying she needed to see the doctor again, straight away.

“My results showed my blood platelet count was up near the two million mark, and when I asked what the usual range was, it was 450, so it was massively out of the normal range.

“They said, ‘look, it’s in line with essential thrombocythaemia’ and sent me to the emergency department, to see whether I needed to see someone urgently about it.”

Jennie was told there was nothing they could do at that time, and to go home, take some aspirin and the haematology department would be in touch.

“Within a week, they did some very thorough work and checked all my levels. I had an ultrasound of my spleen, a CT scan of my brain, and a bone marrow biopsy, before I started any medication.

“Then I went on hydroxyurea, initially, to help control my platelet count.

“The bone marrow biopsy actually revealed the early stages of myelofibrosis (MF). I’ve got some scarring on my bone marrow,” said Jennie.

Her diagnosis was a combination of MF and ET and she began seeing her haematologist every fortnight and had regular blood tests.

“The platelets started responding straight away and they’ve gradually come down, but not ever to within the normal range.

“When I first found out I had this condition, I was told MPN normally affected people in their 60s or older,” said Jennie who was 35 at the time.

“I wondered what it meant for me as a younger person and somebody who wanted to continue with having more children. I didn’t know what the impact was going to be, so obviously it was a little bit scary.

“I remember that day coming back from the doctors and just being in shock and thinking, gosh, what does this all mean? You think about your own immortality… am I going to be around to watch my children grow up, or my child at that point, and am I going to be able to have more children? You just don’t know.

Jennie Wiggington and her sons
Jennie and her sons, George and Austin, wearing her Metafit Footscray business hoodies

“And I’d never heard of it [MPN] before and didn’t know anything about the condition.

“So, you’re just really shocked by it because it took me out of the blue. Suddenly I’m being told I’ve got a blood cancer, and not knowing what the future held,” said Jennie.

“I’m someone who likes to keep fit but I had to balance that as I was concerned because I was at risk of blood clots because of my high platelets.

“Once my blood platelets came down more into range, I felt more comfortable. I was taking aspirin to thin the blood, and the doctor said it was good to keep exercising and moving.”

When first diagnosed, Jennie mentioned to her haematologist that she had been unsuccessful in falling pregnant again. She was told it might be due to her blood condition and to stop trying for the moment.

“I went to a fertility specialist who talked about having cancer as a young person and the implications of different treatments and how they can impact on your fertility,” said Jennie.

“We talked through options about the possibility of freezing eggs, or IVF, stuff like that, but as we were not at that point, we would go down the natural route if we could and see how things go.”

Hydroxyurea successfully reduced Jennie’s platelet count over six months, from May to October 2015. Then, in November, she transitioned to interferon over three months before stopping hydroxyurea because of the implications that can have on a developing baby.

She initially injected herself with the interferon three times a week before the dosage was increased to five times a week.

“When you start that medication, often you get flu-like symptoms and start to feel quite groggy, but luckily it only lasted for the first few days. Your body adjusts and gets used to it. I was advised to take Panadol to help with any kind of aches.

“It was Roferon-A, not the pegylated one, I didn’t move on to that until later,” said Jennie.

“Before we could start trying to fall pregnant again, I had to be clear of the hydroxyurea for three months, so it had all got out of my system.

Jennie Wiggington with her medal for Melbourne Marathon
Jennie competed in the Melbourne Marathon (Half) in October 2019

“I remember the doctors saying not much is known about pregnancy and MPN because not that many people of childbearing age have the condition.

“So it was kind of, ‘we know that you can’t have children on hydroxyurea and we believe interferon is the safest option for you, and the research shows that there’s not an impact on the baby’.

“I guess they don’t know 100 per cent, but it’s the safest form of treatment whilst you’re pregnant.

“But there were still risks of clotting and haemorrhaging and the placenta coming away from the womb. I was told that those were the inherent risks around having another baby.

“It was hard because we’d had our son. Did we just say, ‘that’s it, we’re done, and we’re not going to risk it?’ because the risks weren’t just for the baby, but potentially for me as well because of the blood clotting.

“We just took each step at a time and listened to the advice from the specialist doctors,” said Jennie.

“We got pregnant again in August 2016. Because of my blood condition, it was deemed as a high-risk pregnancy and I was under the care of specialist doctors.

“I was getting the best advice and care with lots of support, regular appointments, and monitoring.

“They picked up very early on, when I was about six weeks pregnant, that my thyroid levels were through the floor and in the danger zone for the baby developmentally.

“I had to go on thyroxine. They don’t know if the interferon had impacted my thyroid and damaged it, which it can apparently do, or whether I got Hashimoto’s disease, which is hereditary – my sister and my aunty both have it. Or it could have been brought on by pregnancy,” said Jennie, who continues to take thyroxine.

Mid-way through her pregnancy, with the blessing of her doctors, the Wiggintons went back to England for a month and a family Christmas. Jennie and Russell had migrated to Australia in 2010.

“I just had to make sure I had all the medications with me,” said Jennie.

She took an anticoagulant (blood thinner) before, during and after each flight, to minimise the risk of blood clots, and she drank lots of water.

Wiggington Family in New Zealand
Holidaying in New Zealand in July 2018 – Russell and Jennie with George and Austin

All in all, Jennie’s pregnancy went well, and her obstetrician said she had done ‘amazingly well’. To minimise the risks during labour, the decision was made to induce her in the 39th week.

“Then nothing happened for 24 hours and, as I wasn’t dilated enough and the baby’s heart rate dropped, they did an emergency caesarean.

“He had some low blood sugar, because he was quite little when he was born, and had to go into special care for a few days until his blood sugar stabilised.”

George was born in May 2017, “a fine, healthy baby boy”, and Jennie and Russell, who had always hoped to have two children, and feeling lucky nothing major had happened, decided “enough’s enough, we’ll finish on a good note”.

Jennie continued her interferon medication until she heard that pegylated interferon [Pegasys®] become available on the Pharmaceutical Benefits Scheme.

“I went straight to my haematologist saying, ‘I’d like to try this’. I started on a low dose and that’s what I’ve been on since.

“It’s been great because it means now I only have to inject once a week rather than five days a week and have no ill effects from it. It’s just been a revelation, really, in terms of treatment for the illness.

“I never had any horrible side-effects on Roferon-A,” said Jennie, but she used to have “really bad itching” which was worse in winter when her skin was dry.

“I’d scratch so bad I’d bleed on my legs and on my tummy, but that’s gone for me now. I don’t know if it’s the pegylated interferon, or if it’s changing hormones or the environment.”

Jennie self-injects the pegylated interferon into the fatty subcutaneous area around her stomach.

“Sometimes, I inject into my leg, just to give my tummy a bit of a rest.”

Over the course of Jennie’s experience with MPN she has gone from seeing her haematologist every two weeks, to once a month, then six-weekly, and now it’s every three months.

When she spoke to MPN News Jennie had just received her latest results.

“My platelets were the lowest since I was diagnosed and only very slightly above the normal range which is really good.”

In June, Jennie asked to have her bone marrow tested again even though this is not usually done unless there is a change in presentation or bloods.

“Overall, it looks like the scarring has got worse since five years ago, but he [her haematologist] said it’s hard to decipher if it’s the progression of the disease or the impact of the interferon.

“As everything else looks good and there are no blast cells present, the decision was to carry on as normal.

“The only other options would be to go on ruxolitinib [Jakavi®] or a stem cell transplant but we decided, neither at this point.”

Jennie said she had been affected by “really bad headaches again”.

“Automatically, I go back to the worse-case scenario in my head, what’s wrong, something’s happening. Am I going to have a stroke because I have a blood clot or is the condition getting worse?”

“I’ve got a really good GP. She’s really thorough. She sent me for MRIs and referred me to the neurologist.

“I think it’s just about trying to keep a level head and also listening to your body.

“We’ve recently moved and had a lot of changes and I think it’s also about being kind to yourself and taking a bit of time out.”

In early-July, the family moved from Melbourne to rural Woodend where they have a “massive garden and a view of Mt Macedon”, and there’s been a new addition to the family, Monty, a kelpie puppy.

Since COVID-19, Jennie has worked her three days of social work a week from home rather than driving the hour into Melbourne, and she has moved her fitness business online and is training her clients from a distance. She keeps fit by running most days “for fitness and to clear your head”.

“I don’t let it [MPN] control me or dictate who I am or what I am, but sometimes I need to remember that I have this condition, and I do need to listen to my body and just slow down and rest, if my body’s telling me to,” she said.

But, as a mum with two young kids, running a family, and working, Jennie said, “it’s a necessity to keep going”.

“I do what I can to stay healthy, and ultimately my family is my inspiration.

“I want to be here for as long as I can to be with them and look after them and watch them grow up.

“It’s good to know that support is there if I need it.”

Jennie has joined the Leukaemia Foundation’s coffee mornings and been to the blood cancer conference in Melbourne a couple of times, although with a young family that’s not always easy.

“It takes quite a lot of effort for me to get to those meetings. I need to take time off work and find childcare,” she said.

“I’m on a few of the Facebook support groups. There are people in a similar situation to me, young, and wanting to talk about having babies with the condition. I’ve been able to share my knowledge and experience.

“It’s nice to know that you’re not out there on your own, especially when initially diagnosed, and that there are other people experiencing similar difficulties and that there are great support networks available to help and reassure each other.”

Clinical trials critical to finding curative therapies for MPN

Clinical trials critical to finding curative therapies for MPN

Dr David Ross and others in Tokyo
Associate Professor David Ross, centre, at a conference in Tokyo

Associate Professor David Ross is a clinical and laboratory haematologist who has always had an interest in MPN.  His clinical PhD scholarship in CML, monitoring residual disease, was funded by the Leukaemia Foundation. He is Head of the Clinical Trials Unit at the Royal Adelaide Hospital and Director of the South Australian Cancer Research Bio Bank. In this comprehensive interview he discusses everything from research, current therapies, clinical trials, diagnosis, prognosis, incidence, and more, and says, “it’s a very exciting time in MPN”.

After “almost nothing” by way of new treatments for 20 or 30 years, “there’s just been this massive explosion of clinical trial activity in MPN, said Associate Professor David Ross.

“We’ve gone from a situation where there were basically no new treatments, to one where a dozen drugs have been in clinical trials over the past few years.”

But one of the big issues in MPN remains.

“In CML, we have drugs like imatinib that essentially turn the disease off and, for most patients, ensure that it will never transform to a more aggressive phase, and the patient will never die from leukaemia,” explained Dr Ross.

“Therapies have improved some of the clinical manifestations in MPN, but the drug treatments available don’t change the long-term outcome of the disease.”

Does he see this changing?

“Look, I think it will. There’s been a huge amount of research on MPN in the last 10 or 20 years.”

Dr Ross said his holy grail is “to have a treatment for MPN that is curative, to be able to give someone a course of treatment that completely gets rid of the disease, gets rid of future risk, gets rid of any current symptoms or problems”.

Dr David Ross
Dr David Ross

JAK2 mutation – the key discovery

The JAK2 mutation is found in almost everyone with polycythaemia (rubra) vera (PV) and more than half of people with essential thrombocythaemia (ET) and myelofibrosis (MF).

“That key discovery, first published in 2005, has given scientific insights into these diseases that has spurred a lot of research and development,” said Dr Ross.

“Then there’s the calreticulin (CALR) mutation, found in about a third of patients with ET and MF. This second most common mutation was only discovered in 2013.

“That extra scientific information is a clue for academic researchers and drug companies to start understanding the disease and looking for drugs that can target those particular pathways.

“That’s where the JAK inhibitors came from, like ruxolitinib (Jakavi®), but as people better understand what CALR does, and what JAK2 does, and what MPL [another MPN mutation] does, they may find other targets that might be more effective.”

Next generation sequencing

Another important development was ‘next generation’ sequencing (NGS). Traditional sequencing looks at one small section of a single gene. NGS looks at many different sequences, often in many different genes, all at the same time.

“NGS panels are available for various blood diseases with lots of different mutations. These may test five or six genes, sometimes 30 or 40 genes, so a single test will give you a large amount of information,” said Dr Ross.

“There’s an increased use of sequencing panels to look for not only JAK2, MPL, and CALR, but also other mutations that may be associated with higher risk disease and that currently are most relevant for MF. They are IDH1, IDH2, ASXL1, EZH2, U2AF1, and SRSF2.

“The presence of a mutation in one of those genes increases the risk of MF, and for a small group of patients that’s really essential information, used in guiding transplant decisions.

Sequencing panels may also be useful to clarify the diagnosis.

Dr Ross went on to explain that if someone is intermediate risk, but doesn’t have any bad mutations, that might downgrade that person to being low-risk. Whereas, if someone is intermediate risk and has one or two of those mutations, that might push that person up into a high-risk group where the life expectancy might be only two or three years, and convert them from a watch and wait approach to going straight to a bone marrow transplant.

Dr Ross said this panel test was not currently funded by the Federal government.

“As is usually the case, the Medicare rebate for the test lags years behind research and clinical practice, so individual hospitals are paying for it, or sometimes individual patients pay to have it done privately.”

He said the cost varied from $400 for a small panel looking at the most common mutations in a particular gene, up to $1500 for a more extensive panel that sequenced 30-40 genes.

“But when you think that a bone marrow transplant might cost quarter of a million or half a million dollars, this is a trivial amount of money.”

Each state has different rules about getting tests done.

“In South Australia, everyone with MF who’s been discussed for transplantation would get this done; that’s a small number of patients out of the total MPN population, because MF is the rarest of the MPNs and only 25% or less of MF patients will be transplant-eligible.”

Clinical trials in MPN

Most recent studies have been for myelofibrosis, reflecting it being the MPN with the highest need.

Ruxolitinib was the original JAK inhibitor. Several studies have explored other JAK inhibitors (fedratinib, pacritinib, and momelotinib) on their own, or comparing them to ruxolitinib.

“Different companies are looking to see if one of the newer JAK inhibitors works after ruxolitinib has failed, or offers advantages over ruxolitinib in certain patients,” said Dr Ross.

“For instance, there is some hope that pacritinib might be better in people with a low platelet count, and momelotinib might be better in people with a low haemoglobin.

“Neither has been proven, but these are the questions that are being looked at in clinical trials.

“We currently have a momelotinib study [called Momentum] that is recruiting patients with myelofibrosis who are anaemic.

“The ‘mel’ in the name is because it was originally developed in Melbourne,” he explained.

“It’s already been used in hundreds of patients, so we know that it works.

“Most people on ruxolitinib have a modest drop in haemoglobin; they become more anaemic. It’s been observed that with momelotinib, the drop in haemoglobin is less, and some patients have an improvement in anaemia.

“So, whether momelotinib will offer an advantage specifically in the subgroup of people with myelofibrosis who are anaemic is being explored,” said Dr Ross.

Momelotinib and ruxolitinib both inhibit JAK1 and JAK2. Another study testing fedratinib will try to answer the question of whether there is some advantage to a pure JAK2 inhibitor [it doesn’t inhibit JAK1]. This study will recruit MF patients who have a had a suboptimal response on ruxolitinib, but  is currently on hold due to COVID-19.

The Kartos study opened recently. KRT-232 is an MDM2 inhibitor being tested in MF patients who have failed on ruxolitinib therapy. Dr Ross said MDM2 was involved in the P53 pathway, which is important in lots of different cancers. It’s a quality control pathway within the cell that senses DNA damage and causes the cell to undergo apoptosis [cell death] if there has been DNA damage.

“These are all international studies that include Australian sites,” said Dr Ross.

And there are other drugs in completely different classes that have different mechanisms of action that have been tried in early phase studies.

Australians were among the first patients enrolled on an ongoing study of bomedemstat that inhibits an epigenetic enzyme involved in controlling blood cell production.

“It’s a tablet and it’s shown some improvements in symptoms and spleen size and is generally quite well tolerated,” said Dr Ross.

An initial study of ruxolitinib combined with another class of drugs, called BET inhibitors, showed some encouraging responses. Now a larger study is in the planning stage and may open in Australia in the next six months.

Experimental data suggests navitoclax, which is related to venetoclax, and inhibits another member of the BCL-2 family, may be useful in MF, and luspatercept is being explored to see if it improves anaemia in MF.

The ADORE study is open at several sites for Australian MF patients who are on ruxolitinib and are anaemic. It is a Phase I platform study looking at a series of experimental drugs being added to ruxolitinib. A small number of patients will try each combination and then the results will be reviewed to decide which combination is the most promising, to take it to a bigger study.

“So, it’s a ‘pick a winner’ study,” said Dr Ross.

Studies in PV and ET

Dr Ross said that the first clinical trial in Adelaide for PV closed recently. It was using another MDM2 inhibitor called idasanutlin, “and it definitely works in some people who failed standard treatment”. The study closed due to toxicity concerns.

“The main issue was nausea. You can imagine that if you’ve got PV and you’re going to live with the disease for 10 or 20 years, having a drug that causes nausea for a week every month is not very good for quality of life.”

He also is “quite excited” about an upcoming ET study, also using bomedemstat. The opening of this study has also been delayed by COVID-19 but is expected in late-2020.

“It will be our first ever ET study in Adelaide.”  

“Because we’ve already had experience with that drug, we know that its safety profile is pretty good, so I’m optimistic about that.

“It will be for people who have been resistant to, or intolerant of hydroxyurea, which is the standard treatment for most people with ET.”

Ask about studies for you

“There are many studies for myelofibrosis at the moment – we’ve currently got four in South Australia – and a lot of the time they’re competing for the same rare patient population,” said Dr Ross.

“For companies to test their drugs, they need more patients.

“If we can’t enrol patients in clinical trials, it slows down the development of a drug and means that our patients won’t get normal access to the drugs because it takes longer to do the study properly.

“This is the problem of a rare disease.”

Dr Ross urged patients with MF to be proactive in asking their clinicians about clinical trial options in their city.

“A lot of these studies are open in only one hospital or maybe two hospitals in a bigger city. We need people to be referred to sites where a study is open, so we can put people on them,” he said.

“And they can look on the ClinTrial Refer app or website to see whether there’s anything that meets their particular circumstances.

“A lot of these studies are looking for only a few patients in each hospital, who meet very specific criteria, but if there are five or six studies, there is room for a lot of patients,” said Dr Ross.

The prime target population in MF are those patients on ruxolitinib or who have been on ruxolitinib and have not had an optimal response, and the main focus of these studies is to improve on the benefits already seen with ruxolitinib.

MPN is different from other blood cancers

MPN is a blood cancer, said Dr Ross, “but the way it behaves is completely different from lymphoma or leukaemia, so many people with MPN can go undiagnosed”.

“What sets ET and PV apart from other diseases, is that many people have either no symptoms or vague symptoms, like tiredness, together with blood count abnormalities, which means they may go undiagnosed for some time,” he explained.

“They’re often overlooked for a long period of time. That’s one of the standout features of ET and PV, and the main risk is bleeding and clotting (venous or arterial thrombosis) that can come out of the blue in people who didn’t know that they had an MPN.

“That makes it quite different from most other cancers. You’re not treating a tumour or trying to clear out the leukaemic cells, you’re mostly trying to protect the patient from having a clotting or bleeding episode.

“And the longer you leave it, the more chance there is of having a clot.

“What we know is that some people turn up and they have a clot that could potentially have been prevented if the diagnosis had been made earlier. So, a significant fraction of people with MPN will first be diagnosed when they present with a blood clot or a stroke or a heart attack.

“Sometimes, in those cases, we see someone who comes in having had a high platelet count for three years, and nobody’s done anything about it. So possibly, if that person had received appropriate treatment, he or she might never have had that clot,” said Dr Ross.

He cited the findings of a colleague with a long-standing interest in MPN, Dr Cecily Forsyth. She went back through the records at her centre, at Gosford (NSW), looking at people with a diagnosis of MPN and tracked their haemoglobin, white blood cell, and platelet counts.

“One of the patients had a high platelet count for 20 years before a diagnosis was made,” said Dr Ross.

“The estimated risk of having a clot is about 2% per year for ET, and maybe about five or 10% for PV. Many people, just by chance, will go years without having any problems, but someone’s got to be the one who’s unlucky.

“If someone has blood tests for other reasons and a mildly elevated platelet count is noted, it would often be disregarded if the person is otherwise well.

“In most laboratories, a normal platelet count is 150-450. In fact, your platelet count might be 300 or 200, but the top of the normal range is commonly close to 450.”

What complicates things is that if you are unwell, for instance if you have a chest infection, or if you are iron deficient, your platelet count might go up.

“In young women, in particular, it’s quite common to be iron deficient due to periods or pregnancy, so a slightly high blood count may be disregarded.

“And in an older person who’s got arthritis or other chronic health problems, a slightly high platelet count might be put down to inflammation.

“It’s when there’s a sustained elevation, with no obvious cause, that someone needs to think, ‘well, actually, this has been present two or three times. It needs to be looked into’.”

Myelofibrosis and stem cell transplantation

Dr Ross said myelofibrosis (MF) is completely different from ET and PV.

“Most people with MF feel unwell. They often have severe tiredness, itching, sometimes night sweats, and discomfort from enlargement of the spleen,” he said.

“The main aim of treatment is to lessen the severity of those symptoms, improve quality of life, and make people feel better.

“And, because MF is a more serious disease with a shorter life expectancy, we do bone marrow transplantation for higher risk MF patients who are young and fit, in whom the risks of transplantation are warranted.

“The age limit for transplantation has been continually creeping up over the past decades. Now, for most sites around the country, it’s up to age 70.

“Unfortunately, the risks associated with the transplant rise steeply once you’re above 50 [years old], and the chances of dying from the transplant if you’re nearly 70 are pretty high. The average age at diagnosis of MF is also around 70, so we don’t do very many transplants for myelofibrosis, but it is a potentially curative treatment,” said A/Prof. Ross.

Transplantation is not used for ET or PV because the risk is rarely justified. The treatments for those diseases are about controlling the blood counts and reducing the risk of clotting, but they’re not eradicating the disease or reducing the risk of future progression.

Diagnosis of MPN and its importance

MPN diagnoses are currently based on blood counts, bone marrow appearance, and clinical features, such as itching, sweating and spleen enlargement, said Dr Ross.

“It’s an old-fashioned classification system.”

He said Professor Tony Green’s group in the UK published a “highly influential” paper in the New England Journal on the results a large group of 2000 patients whose MPN was classified based on the results of genomic sequencing.

“They looked at patterns of mutation and showed that the behaviour of the disease and long-term survival could be predicted with some accuracy just by looking at the genes without the traditional pathological classification,” said Dr Ross.

“This hasn’t yet changed the way that we diagnose MPN, but it has emphasised the fact that you can get a lot of useful clinical and biological information from extended sequencing that may add to our old-fashioned classification system.

“And, in some cases where the bone marrow appearance is difficult to interpret, and one pathologist might think it’s ET, and another thinks it’s early myelofibrosis, looking at the sequencing for these difficult-to-classify cases might tell you, well, actually this is more likely to be MF, or actually, this is more likely to be PV.

“The biggest distinction is to identify early myelofibrosis and that’s important because the life expectancy is much shorter, and transplantation might be an option.

“And drug access is determined by having a biopsy that says you have myelofibrosis. You can only get ruxolitinib on the Pharmaceutical Benefits Scheme (PBS) if you have myelofibrosis; you can’t get if you’ve got PV or ET.

“It has been proven that ruxolitinib is effective in hydroxyurea-resistant and -intolerant PV, but it hasn’t been funded [by the PBS] because of the cost.”

“At the moment ET and PV are treated similarly. They both usually get hydroxyurea or interferon plus aspirin.

“The difference is that in PV, we aim to keep the hematocrit, which is a measure of haemoglobin, below 45%, as well as the platelet count and white cell count in the normal range. Whereas in ET, we only look at the platelet and white cell counts.

“Lots of drugs are being investigated at the moment and if any of those make it to clinical practice, the implication of making an accurate diagnosis will become more important,” said Dr Ross.

Incidence and prevalence of MPN

A paper published last year in the American Journal of Hematology based on epidemiology work by Professor Peter Baade reported on the latest available statistics on incidence, prevalence, and survival of MPN in Australia.

“This showed there are 23 cases of MPN per million population per year, so it’s a pretty uncommon disease, but because many people with MPN will live for many years, the prevalence is relatively higher, considering the low frequency of diagnosis,” explained Dr Ross.

“For instance, somebody with ET might live for 20-30 years, whereas for many cancers the survival will be much shorter.

“According to that study, new diagnoses of PV and ET were roughly equal; at about nine per million per year, and primary myelofibrosis is the rarest at about five per million per year.”

Regarding age at diagnosis, Dr Ross said the average age of diagnosis for all MPNs was 68. The oldest cohort, with an average age of 72 years, were those with primary myelofibrosis, and the youngest was ET at 66 years, closely followed by PV at 67.

However, he said, “there’s a tail of younger patients”.

“We see people in their 20s with ET, whereas myelofibrosis is rare below the age of 40.”

“The cause of MPN in most cases is unknown. If you have a family member with MPN, your risk of getting an MPN is increased about five-fold compared to the general population.

“Although it’s not an inherited disease, there’s an inherited risk component. We do occasionally see brothers and sisters or parents and children that both have MPN.”

Dr Ross said there weren’t any known strong risk factors, although there is an increased risk with exposure to radiation and, rarely, to industrial chemicals.

“No-one has ever done a proper, large epidemiological study of MPN risk factors.”

Disease progression and risk

Dr Ross said ET and PV can both turn into MF.

“It’s generally estimated to be something like 20-30% lifetime risk, but that might depend on the age at diagnosis.

“If you’re diagnosed [with ET or PV] at 75, you may never get myelofibrosis. But if you are diagnosed at 30, your risk of getting myelofibrosis might be substantially higher because you’re potentially going to live for another 50 years.

“All three diseases can turn into acute myeloid leukaemia (AML).”

However, Dr Ross said the risk of AML for ET patients is very low, around 2%, and 5% for PV, so it is rare.

“Every now and again it happens, and it is a shock for those people.”

For MF, the risk of AML is 20-30%.

*  Dr David Ross received a Leukaemia Foundation clinical PhD scholarship (January 2006-January 2009, $120,000) for his research project, Characterisation of persistent CML cells in patients treated with ABL kinase inhibitors.

For information about any of the studies mentioned, download the Clinical Refer app on your smartphone and search using the name of a drug or study.