Bone Marrow Transplant for Myelofibrosis

What is a Bone Marrow Transplant?

For allogeneic hematopoietic cell transplantation (otherwise known as a bone marrow or stem cell transplant), hematopoietic (blood-forming) stem cells are transferred from a donor to the patient, essentially replacing defective stem cells with healthy ones. Before the stem cell infusion, the patient receives high doses of chemotherapy and/or radiation therapy to eradicate diseased bone marrow.

Allogeneic hematopoietic cell is currently the only treatment approach in MF that is potentially curative, with a substantial number of patients experiencing complete hematological, histological and molecular remission. However, such therapy is complicated by relatively high treatment-related mortality and morbidity. 

Who is a candidate?

Of the MPNs, only those diagnosed with myelofibrosis would be considered for a transplant. Within that population, only a subset of people will be healthy enough to undergo a transplant. Patients need to carefully discuss whether or not they may be eligible for such a procedure, given the complexity and possibility for complications. Since age and the presence of other diseases elevates the risks associated with ASCT, the decision to pursue ASCT should take into consideration the patient’s risk tolerance and transplant-related prognostic score (a rough measure of likely success, based on factors specific to the patient and the transplant).

Paying for Transplant

Accessing this treatment is further complicated for those on Medicare. Medicare does not provide coverage for transplants to treat myelofibrosis. There are several groups working to reverse this position so that the people who need it have access to this potentially curative treatment. We will report back as soon as this is resolved. In the meantime, if you are facing a transplant and are on Medicare you should let your hospital know ASAP that this is an issue so that you can work with their financial counselors on alternatives for you.

Read Stories of Others Who have Undergone Transplant:

Sources:

Hematopoietic Cell Transplantation as Curative Therapy for Idiopathic Myelofibrosis, Advanced Polycythemia Vera, and Essential Thrombocythemia. H. Joachim Deeg et al. Biology of Blood and Marrow Transplantation,  March 2007Volume 13, Issue 3, Pages 355–365 http://www.bbmt.org/article/S1083-8791(06)00754-3/abstract

Allogeneic hematopoietic cell transplantation versus drugs in myelofibrosis: the risk-benefit balancing act. A. Tefferi. Bone Marrow Transplantation (2010) 45, 419–421; doi:10.1038/bmt.2009.193 http://www.nature.com/bmt/journal/v45/n3/full/bmt2009193a.html#bib13

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