Jamile Shammo, MD:Goals of therapy in myelofibrosis evolve around a patient’s presentation. Exactly what are their symptoms, what is compromising their quality of life? In this situation, clearly, anemia is one item that we need to think about improving. We also want to improve symptoms of splenomegaly, as well as improving the symptoms of night sweats that the patient is dealing with because these tend to be relatively difficult to manage as you evolve with the disease. This is a relentless type of a problem that patients deal with. And so, I think management will be centered around improving the cytopenias, controlling splenomegaly, and improving the constitutional symptoms.
Patients who have low- to intermediate-risk disease tend to have better survival. Essentially, the IPSS risk categorization gave patients who have 0 score, which is considered low-risk, a median survival of around 11 years. So, you can contrast that with the high-risk disease case that we have talked about, and you can see the tremendous difference. For people who have intermediate-1 risk myelofibrosis, their median survival is on the order of 8 years. So, you can see how important it is to actually prognosticate as you make a diagnosis. And, importantly, the IPSS is something that we need to utilize at the time of diagnosis, and you cannot really redo it or reutilize it, say, 2 or 3 years later.
Myelofibrosis is not a very good disease to have. Taken altogether, people who have myelofibrosis have a median survival of about 6 years. Clearly, that has taken into account the low-risk and the high-risk disease, so that gives you the 6-year mark. Unfortunately, this is not a very common, and in order to be able to appreciate the variability from one patient to the next, you need to have a reasonable number of patients to kind of gain experience and get the flavor of each different case. But because the disease is not very common, I suspect that that may be one reason why appreciating the seriousness of this illness may not necessarily take place.
Transcript edited for clarity.
June 2016
Rossetti Reviews Myelofibrosis Risk Stratification and Outcome Data for Pacritinib
July 17th 2024During a Case-Based Roundtable® event, James M. Rossetti, DO, discussed the role of risk scoring and stratification tools and treatment for a patient with declining hemoglobin and platelet counts due to primary myelofibrosis.
Read More
Phase 3 VERIFY Trial Investigates Rusfertide’s Potential in Polycythemia Vera
July 16th 2024In an interview, Aniket Bankar, MD, discussed the background, design, and goals of the phase 3 VERIFY trial of the hepcidin mimetic rusfertide for the treatment of patients with polycythemia vera.
Read More
Ruxolitinib Treatment in Myelofibrosis Still Effective, Regardless of Anemia
June 21st 2024New or worsening anemia did not appear to reduce the clinical benefit of ruxolitinib in myelofibrosis patients, and the median overall survival was similar between patients with and without new or worsening anemia.
Read More
Challenging Disease Features of Myelofibrosis Lead to Use of JAK Inhibitors
June 17th 2024During a Case-Based Roundtable® event, Kristen Pettit, MD, moderated a discussion on which disease features of myelofibrosis are most challenging and when to use JAK inhibitors in the first article of a 2-part series.
Read More