Key Considerations for Assessing Risk and Treating Myelofibrosis


Raajit Rampal, MD, PhD, discusses his recommended approach to assessing and treating patients with myelofibrosis.

Raajit Rampal, MD, PhD, hematologic oncologist at Memorial Sloan Kettering Cancer Center, discusses his recommended approach to assessing and treating patients with myelofibrosis.

Rampal says the first step in addressing a patient diagnosed with myelofibrosis is to determine whether their disease is latent or if is more rapid and has the potential to progress to acute myeloid leukemia. Some patients may live without symptoms for years and not require treatment.

Risk stratification is extremely important in this setting. It is based on many prognostic factors including expression of symptoms, severe or moderate anemia, enlarged spleen, and the presence of certain genetic markers. According to Rampal, risk assessment will guide the next steps for therapy.

Once physicians assess risk, they can consider treatment including Janus kinase (JAK) inhibitors such as ruxolitinib (Jakafi) or fedratinib (Inrebic). Patients at high risk of disease progression should also be evaluated for eligibility for allogeneic stem cell transplantation based on age, performance status, and other factors. Rampal says that at the time that patients start requiring treatment, physicians should consider whether to refer them for transplantation. Bridging therapy may help decrease marrow blasts to enable transplantation.


0:08 | When dealing with a patient with myelofibrosis, the first thing to do is to try to figure out where they are in their disease course because patients can have a latent disease that is relatively quiet for maybe a decade or more, and then there are the patients who are going to progress within a year or 2 to leukemia. Using the existing tools that we have to try to figure out where patients are on that spectrum is key.

Then, figuring out where they fall in the risk spectrum, what is that patient's immediate risk of disease progression? What are their needs? What is the patient facing? Are they having symptoms? Is the spleen bothering them? Do they have anemia that is causing symptoms? That will then guide what you do in terms of therapy.

0:52 | For therapy decisions, do they need medications to deal with any of those symptoms, JAK inhibitors or other drugs? Second, are these patients at relatively high risk of their disease progressing? If they are and we think they're a transplant candidate, then they should be seen by a transplanter. I think that anytime you're starting to need to treat the patient, that is a good time to think about talking to the transplant folks.

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