Stem Cell Transplant for Myelofibrosis
What is a Stem Cell Transplant?
There is no single treatment that is effective for all Myelofibrosis sufferers. Each patient has a unique set of symptoms and circumstances that require different treatment options, as prescribed by a doctor. Also, some patients with MF remain symptom-free for many years and may not require immediate treatment. However, anyone who has been diagnosed with MF needs to be monitored over time for signs or symptoms that suggest the disease has worsened.
Allogenic Stem Cell Transplantation is the only curative treatment for MF. ASCT, 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 chemotherapy and/or radiation therapy to eradicate diseased bone marrow. 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 on-line 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.
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).
MPN Forum Roundtable: Stem Cell Transplantation in Myeloproliferative Neoplasms
Stem Cell Transplant Timing Tool
In an effort to improve stem cell transplant outcomes for MF patients, MPNRF supported the creation of the Myelofibrosis Assessment Graphic Internet Calculator (MAGIC). MAGIC 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. SCT Spectrum Transplant Timing tool is available for free on a mobile phone, tablet, and PC.
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.
- 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.
- 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.
- 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.
- 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.
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