Case 1: NCCN Guidelines for Treating Myelofibrosis


Rami Komrokji, MD: Now, once we assess the risk, basically, the next step is really that we’re going to tailor the treatment, and there are 2 things we are thinking of. Obviously, presence of symptoms, and when we think of symptoms, we think what are the big dominant symptoms. Most of the time, we are dealing with splenomegaly and constitutional symptoms, and sometimes we are dealing with cytopenias. Sometimes, those are concomitant, so we try to decide on what is the most important or symptomatic for the patient. The other major issue we are thinking of is the allogeneic stem cell transplant. If the disease risk is high enough, then we’re going to be thinking of allogeneic stem cell transplant.

If we think of patients that may have low-risk disease, if those patients are asymptomatic, I think observation is a very appropriate first step for those patients or to consider a clinical trial. If those patients happen to be symptomatic, then based on the symptoms, particularly if they have splenomegaly and constitutional symptoms, ruxolitinib is probably a reasonable option. Interferon-based treatments can be considered in this early low-risk group, and in my opinion, that’s probably the best subtype of myelofibrosis that would benefit from interferon-based therapy. If patients are intermediate risk, intermediate-1 risk, again, we go by if they are symptomatic. Mostly, we are discussing splenomegaly and constitutional symptoms, then ruxolitinib at this point is standard of care for most of those patients or a clinical trial. If patients are asymptomatic, then we observe them. In a subset of patients with intermediate stress group, if they have other risk features, they are younger, we may entertain or discuss the role of allogeneic stem cell transplant for those patients.

Now, when we move to the intermediate or high-risk group, again, those are the patients we are thinking from the get-go, considering allogeneic stem cell transplant. If they are a candidate, we proceed to allogeneic stem-cell transplant. Sometimes, we will bridge those patients with JAK2 inhibitors because they could have a large spleen, they are symptomatic, and improving their splenomegaly and symptoms prior to transplant may improve their outcome. If patients are not candidates for allogeneic stem cell transplant, then we go again asking the question, what is the predominant symptom and what’s affecting the patients? If it’s the cytopenia, then we go through management of cytopenias, predominantly anemia, with limited options that we have in that area. If the dominant symptoms are again, splenomegaly, or constitutional symptoms, we are going to be considering JAK2 inhibitors. There is a subset of patients that will have thrombocytopenia, that it becomes a little bit more difficult to manage. However, when discussing how to dose ruxolitinib in that group, and nowadays, we have other new approved agents such as fedratinib, that can be considered in that subset of patients with thrombocytopenia.

Transcript edited for clarity.

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