Preventing Transformation and Adverse Events Among Patients With Myelofibrosis

Eytan M Stein, MD, Memorial Sloan Kettering Cancer Center, New York, New York, shares expert insights on preventing transformation and reducing the symptom burden of adverse events among patients with myelofibrosis. 

Oncology Learning Network: What strategies do you recommend for oncologists seeking to prevent transformation from myelofibrosis to acute myeloid leukemia?

I think it's difficult to know how to prevent transformation from myelofibrosis to acute myeloid leukemia. You really need something that is disease-modifying. Right now, most of the drugs—or all the drugs, really—that are approved for the treatment of myelofibrosis are very effective at getting rid of the symptoms of the disease, but don't necessarily prevent transformation. 

I think that's an area of active research and certainly, that's something that we're all hoping comes soon where we can prevent the development of transformation.

What adverse events are especially concerning among patients with myelofibrosis?

Well, the major issue with myelofibrosis, as you know, is that the patients develop failure of their bone marrow, so they develop significant cytopenias, and then the second thing that happens is they can get very severe constitutional symptoms. So, weight loss, a very large spleen, sort of drenching [and] debilitating night sweats, fevers, joint pain, [and] bone pain.

What treatments or strategies do you use to relieve adverse event symptoms?

The[re] are things that I think we've done an okay job with when it comes to relieving the symptoms by giving patients [Janus kinase] (JAK) inhibitors. We actually have 3 JAK inhibitors that are now approved, including ruxolitinib, pacritinib, and momelotinib. 

But, I think that we also need agents that work in different ways and then we also need to think about combinations of agents that hopefully will not only get rid of the symptoms but also be disease-modifying.

Source:

Stein EM, Fathi AT, Harb WA, Results from phase 1 of the MANIFEST clinical trial to evaluate the safety and tolerability of pelabresib in patients with myeloid malignancies. Leuk & Lymph. Published online: January 23, 2024. doi: 10.1080/10428194.2023.2300710