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Clinical trial comparing allogeneic stem cell transplants vs best available non-transplant therapies for myelofibrosis

ALLO-BAT is a new myelofibrosis (MF) clinical trial conducted in collaboration with the Princess Margaret Cancer Centre, Toronto Canada which will compare best available non-transplant therapies with stem cell transplant.

Australian Trial Lead: Assc. Professor Nada Hamad.

Assoc. Prof Nada Hamad, Kinghorn Cancer Centre (Sydney)

Primary myelofibrosis MF is a rare chronic blood disorder diagnosed in an estimated 1 per 100,000 population. In MF normal bone marrow tissue is gradually replaced with a fibrous scar-like material. Over time this destroys the normal bone marrow environment, preventing the production of adequate numbers of red cells, white cells and platelets. This results in anaemia, low platelet counts and the production of blood cells in areas outside the bone marrow for example in the spleen and liver, which become enlarged as a result. Over time, MF leads to progressive bone marrow failure.

There are many new therapies being offered however stem cell transplant is potentially a curative option for MF patients. While is may be curative in some individuals it also a high-risk procedure with an associated mortality rate (death) of up to 50%. While there are many new MF drugs coming into the market transplant remains an important strategy and treatment option for these patients. There is currently no robust evidence on if these new non-transplant therapies have survival or relapse benefits.

The rare nature of this disease means that it is difficult to conduct high quality research into transplant for MF patients in Australia. The international ALLO-BAT trial provides a great opportunity to participate in an international study to the benefit of Australian patients and to build local researcher capacity.