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.

The 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 of 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).

 In an effort to improve stem cell transplant outcomes for MF patients, MPNRF created a Stem Cell Transplant Timing Tool. The SSTT is a portable online tool based on a clinically validated scale. It provides a color signal in response to information entered by a patient, that indicates a risk level and median survival times without a stem cell transplant. A patient can then take what they've learned and use it to facilitate a meaningful dialogue between themselves and their physician about their treatment options. Click here to learn more about the SSTT.

SCT Spectrum Transplant Timing tool is available for free on a mobile phone, tablet, and PC. Click here to learn more and to evaluate your risk level today.

Preparing for a Transplant

Below are 6 presentations recorded at the 2018 BMTinfonet.org Celebrating a Second Chance at Life Survivorship Symposium with a multitude of information about how to prepare yourself both mentally and physically for an upcoming bone marrow transplant.

  1. Your Personal Survivorship Plan: What, Why and How: The presenter in this video, Dr. Linda Burns, will discuss bone marrow transplant survivorship care plans and why they are recommended for patients undergoing a transplant.
  2. Strive to Thrive: How to Protect Your Health after an Autologous Transplant: Dr. Peter McSweeney presents an overview of an autologous transplant and discusses the side effects you can expect to see after the procedure.
  3. Strive to Thrive: How to Protect Your Health after an Allogeneic Transplant: In this video, Dr. Catherine Lee discusses the good and the bad of allogeneic transplants and how you can manage the late effects.
  4. Managing Emotional Challenges after Transplant: Transplant survivor, Dr. Melanie Stachelski, presents on common thoughts and emotions after your transplant and provides resources for support.

Paying for Transplant

As of 2016, Medicare now provides coverage for stem cell transplant for those patients who need one. The coverage is reserved for myelofibrosis patients who have a Dynamic Prognostic Scoring System intermediate-2 or High primary or secondary MF score. They must also agree to participate in a prospective clinical study to evaluate their outcomes vs patients with the same score who are not undergoing a Stem Cell Transplant. 

Read Stories of Others Who have Undergone Transplant:

Read the relevant research articles below about Stem Cell Transplants for MPN patients.

Which Patients with Myelofibrosis Should Receive Allogeneic Stem Cell Transplantation? A Decision Analysis Based on the Systematic Review of 4,341 Patients. Monia Marchetti and Nicolaus Kroeger Blood 2017 Click here to read more.

Transplant Decisions in Patients with Myelofibrosis: Should Mutations Be the Judge? Salit RB1, Deeg HJ2. Biol Blood Marrow Transplant. 2018 Click here to read more

Allogeneic Stem Cell Transplantation for Myelofibrosis in 2012 Donal P. McLornan, et al. British Journal of Haematology 29 March 2012

 

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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