How to Individually Tailor Myelofibrosis Treatment Options

The discovery of the JAK2 gene mutation marked a turning point in the treatment of myeloproliferative neoplasms (MPNs), including myelofibrosis. This breakthrough led to the development of a new class of targeted therapies called JAK inhibitors. These medicines work by blocking the overactive JAK-STAT signaling pathway, which plays a key role in driving disease symptoms and progression.

Today, four JAK inhibitors are approved by the U.S. Food and Drug Administration (FDA) for the treatment of myelofibrosis: Jakafi (ruxolitinib), Inrebic (fedratinib), Vonjo (pacritinib), and Ojjaara (momelotinib). Each drug offers unique benefits and considerations, and understanding how to choose among them can be complex.

To shed light on how physicians tailor these treatments to individual patient needs, as well as how patients can work closely with their care teams to navigate evolving options, CURE spoke with Dr. Prithviraj Bose. He is a professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center in Houston.

CURE: When doctors are deciding on a treatment for myelofibrosis, what factors are most important in tailoring therapy to a patient’s needs?

Bose: Myelofibrosis therapy today is still limited to the four JAK inhibitors. However, we do use some unapproved therapies in [certain] situations. For example, interferon can be used for very early disease when the spleen is not enlarged, symptoms are not prominent and there are no cytopenias, with the hope of disease modification.

Are there treatment options for patients whose main problem is anemia?

Yes, there are some patients, not too many, who present with just anemia as their main [issue]. In that case, we can use therapies directed purely at anemia. But most patients will eventually need JAK inhibitors because they tend to have some degree of splenomegaly and symptoms.

How do doctors choose between the different JAK inhibitors?

That is where we really need to be aware of the strengths and weaknesses of the four approved drugs: Jakafi, Inrebic, Vonjo and Ojjaara. It is important to really evaluate the data [of these agents] and try to tailor it [for each individual patient]. For example, with significant anemia, I would typically reach for Ojjaara. Jakafi, however, has a survival advantage, so sometimes you might choose Jakafi and counteract the anemia with another therapy.

A lot of factors go into this decision-making, but the most important thing is to target what the patient needs the most.

With so many new therapies and combinations being studied, how can patients best work with their care team to understand their treatment options?

MPNs are still rare enough that patients are best advised to see an MPN specialist. While they can certainly work with their local hematologist, it's a good idea, in general, given that these are rare conditions, to see someone who focuses on MPNs. They can give the patient a more nuanced understanding of the different treatment choices we have today, as well as the profusion of clinical trials.

The trials can be a dizzying maze. There are so many trials in so many settings; there are so many designs. I often have patients who have read up on them but who have not perhaps been able to appreciate some key differences in the design, which I don't blame them for, as this is not what they do.

Overall, I think it's really important that patients try to seek care from someone who focuses on this area and is well-versed with everything that's going on.

Transcript has been edited for clarity and conciseness.

Reference

1. “What Does a Blood Cancer Diagnosis Mean?,” by Ryan Scott. CUREtoday; Sept. 5, 2025. https://www.curetoday.com/view/what-does-a-blood-cancer-diagnosis-mean-

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