Goals in Treating Low-Risk Primary Myelofibrosis

Srdan Verstovsek, MD, PhD: Patients with low-risk myelofibrosis have a good survival rate, and the risk of dying is not that high. That means they have many years to live—shorter than expected but still many years. That’s what the risk is all about. When we talk about the low risk, we are talking about the risk of dying. Characteristics utilized to assess the risk of dying include quality of life. Many patients are asymptomatic. We’ll talk about the general systemic symptoms: night sweating, low-grade fevers, weight loss. However, there are patients that may have a big spleen, which is not 1 of the factors, and may have symptomatic splenomegaly. In this situation, one would treat the patients. Why let the patients have any kind of symptoms? There are therefore patients in the low-risk situation who we treat. That would be with JAK inhibitors.

If the patient has intermediate or high-risk disease, then the situation usually changes. The characteristics would then be anemia or there would be high white blood cell count and such factors. But there are highly likely then many more cases of symptomatic disease in that setting. Now, there are still possibilities of having asymptomatic patients, even with the high-risk disease. It is not common, because progressive anemia, progressive increase in the spleen, progressive increase in white blood cell count feature, characteristics of intermediate and high-risk disease, usually come with symptoms. It’s a matter of asking questions. Asking questions is one of the important parts of the every-day communication with the patients in the clinic who have myelofibrosis.

But, if the patient is asymptomatic, I usually don’t treat the patients until they become symptomatic. That may not take too much time. Again, it’s a matter of asking questions. If you ask proper questions, we will discover many patients that we think are asymptomatic that are symptomatic.

The factors that would determine eventually whether I treat or not are anemia. For anemia, anemia drugs. For symptomatic splenomegaly or general systemic symptoms, a JAK inhibitor.

Transcript edited for clarity.