Transplant Therapy in Myelofibrosis


Expert perspectives on how to identify and assess patients with myelofibrosis for transplant therapy.


Aaron Gerds, MD: When thinking of transplantation for myelofibrosis, there are 2 main things to think about. One is disease risk, the other is patient health and comorbidities. We want to identify patients who are otherwise healthy. Transplant is a rigorous process, and patients who have heart disease, liver disease, or kidney disease may not do well with transplantation. The first question to ask is how healthy is your patient. Notice that I didn’t mention age, which is just a number. There’s no upper limit for when patients can get transplants with respect to age. We often think about the early 70s as a rough cutoff, but patients have been successfully transplanted and done very well into their 70s.

The other thing is disease risk. We think about patients with high-risk diseases being more appropriate for transplant. Honestly, no matter what disease you’re looking at, whether you’re looking at myelofibrosis, MDS [myelodysplastic syndrome], AML [acute myeloid leukemia], or whatever category, if the predicted survival as a result of that disease is 5 years or less, then transplant tends to have an advantage. If the predicted survival is greater than 5 years, nontransplant therapy seems to have an advantage over time. There was a recent analysis done in a collaboration with the CIBMTR [Center for International Blood and Bone Marrow Transplant Research] that reiterated that point for myelofibrosis, showing that patients with higher-risk disease showed a significant benefit with transplant where patients with lower-risk disease did not.

Pankit Vachhani, MD: The question of who’s the most appropriate patient for transplant or whether my patient is appropriate for transplant is 1 of the most difficult questions to answer in the field of MPNs [myeloproliferative neoplasms] or myelofibrosis. I start by suggesting that everyone risk stratify their patients with myelofibrosis. I do that with the mutation-based risk-stratification tools; for example, the MIPSS [Mutation-enhanced International Prognostic Scoring System] 2.0 or, in the case of secondary myelofibrosis, the MYSEC-PM [myelofibrosis secondary to polycythemia vera and essential thrombocytopenia] model. Let’s say these tools are unavailable, or you’re not familiar with them, or they don’t have the appropriate genetic mutation data available. Then it’s completely all right to use the DIPSS [Dynamic International Prognostic Scoring System] or the DIPSS+ stratification tools. Once you have the risk-stratification tool data available, identify patients on the higher side. That generally means intermediate-2 or high-risk profile mutation patients. These are patients who have survival expectations of 5 years or less. In those events, if patients are less than the age of 70 or 73 and don’t have any major concerning comorbidities, then refer them to transplant. Transplant is the only known curative treatment available, and these would be the most appropriate patients to send for transplant.

Transplant may not be the most ideal and preferred approach in patients with early risk myelofibrosis, or prefibrotic myelofibrosis. There, the survival expectation is much longer, and one may not want to send these patients to transplant immediately. Instead, treat them and manage them as prefibrotic myelofibrosis or overt myelofibrosis as the case may be. If the situation changes dynamically, reconsider the transplant option. Transplant consultation is never a bad idea. One can always consult, figure out the donor situation, have a patient consult with their transplant colleague to understand the pre- and post-transplant sides of the equation, and then sit on that information. Occasionally intermediate-1-risk patients with high-risk molecular gene mutations—particularly ASXL1 frameshift mutations—are also worthy of considering transplant earlier rather than later. My suggestion, even in those select early cases, is to have at least a transplant consultation.

Transcript edited for clarity.

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