Myelofibrosis: Considerations for Transplant or Surgery

Transcript:Kim-Hien T. Dao, DO, PhD: When we look at a patient and try to determine whether they’re a good candidate for allogeneic stem cell transplant, we look at the patient’s comorbidities and also their risk group. Because the procedure is associated with treatment-related mortality, we look carefully at that the disease is high risk enough to warrant undertaking those risks. And so, performing the DIPSS is important to make that determination of whether transplant is a good option for the patient. In terms of whether the patient is a candidate for transplant, again, we look at their organ function and their comorbidities and make sure that the procedure would be a successful procedure and that the patient is healthy enough to undergo such a procedure. And then, we also finally consider whether there’s a good donor option because some patients don’t have good donor options. So, that is also a factor in considering whether we should proceed with allogeneic stem cell transplant.

In determining the timing of when to refer patients for allogeneic stem cell transplant consultation, I usually consider it up front for patients with intermediate-2 disease or for high-risk groups in myelofibrosis. This will allow the patient to meet the transplant physician and talk about the treatment procedure hopefully well ahead before they need the procedure. So, this is more of an introduction to the idea of transplantation to treat their myelofibrosis and potential curative options. But I also consider referring patients for transplantation consult when I see some changes in their disease. If they didn’t meet intermediate-2 or high-risk features, but I see some changes that I’m concerned about—for example, escalating symptoms, increasing cytopenias, or changes in their genetic markers—I will make that referral for a discussion of transplantation in their future.

It’s very important to reassess disease status in a patient because their diseases do change over time, and some of the parameters that we look at will gradually change over time and change their risk group. For some of the patients that are potentially candidates for allogeneic stem cell transplant, it’s very important, because if they are developing high-risk features, this treatment might be the best route to take when their disease changes from low- or intermediate-1 to high-risk features.

A question that often comes up is whether we should be repeating bone marrow biopsies on a regular interview. For my approach on this, I do this on an individual basis. Certainly, if a patient is relatively stable and has no change in counts or symptoms, then I don’t necessarily repeat the bone marrow biopsy on regular intervals. However, if I suspect disease progression, such as change in blood counts, decreasing symptoms, or a leukoerythroblastic blood smear, then I will consider a bone marrow biopsy to reevaluate the stage of the disease.

The role of splenectomy in myelofibrosis is very controversial and must be individualized for the patient. The procedure itself is associated with a very significant morbidity and mortality rate. So, this is a decision that’s not done lightly and should be done at a center that does it frequently with good outcomes. We sometimes go to splenectomy for some patients because their symptoms are uncontrolled. We think that the splenectomy might help their symptoms, but also we sometimes do it because there’s hypersplenism or sequestration of blood cell counts. And by removing the spleen, sometimes we see anemia improvement in a low number of patients. And so, there are various reasons to consider splenectomy, but, again, this has to be done with a very careful risk-benefit discussion. Then, in terms of splenectomy before transplant, this area is also controversial, but, overall, the data are conflicting on whether this is needed before allogeneic stem cell transplant. Currently, this is also individualized because it’s not clear whether splenectomy improves engraftment of the allograft procedure.

Transcript Edited for Clarity