Dr Halpern on Addressing Unmet Needs in Myelofibrosis

Anna B. Halpern, MD, physician, assistant professor, Clinical Research Division, Fred Hutch, assistant professor, hematology, University of Washington School of Medicine, discusses ongoing efforts to address unmet needs in patients with primary myelofibrosis.

One prominent unmet medical need frequently observed in clinical practice is anemia, Halpern begins. Anemia, which is a condition characterized by a deficiency of red blood cells, can emerge as a result of specific diseases or as an adverse effect associated with treatments like JAK inhibitors, Halpern explains. For example, the kinase inhibitor ruxolitinib (Jakafi) disrupts erythropoietin signaling in the JAK-STAT pathway, which is crucial in the production of red blood cells, she adds.

The management of anemia stemming from both the disease itself and ruxolitinib treatment poses a substantial challenge, as effective treatment options in this space are difficult to develop, Halpern continues. This challenge is amplified by the fact that anemia is associated with a decline in the quality of life (QOL), Halpern says. Patients with myelofibrosis also contend with other factors that impact their QOL, such as inflammation and cytokine imbalances, Halpern adds. However, many of these features can be managed using JAK inhibitors, she notes.

Another significant unmet need in primary myelofibrosis is the management of high-risk disease, including the prevention of disease progression, Halpern states. Disease progression and worsening prognosis is typically signaled by an increase in anemia, circulating blasts, thrombo-hemorrhagic complications, and the presence of cytogenetic and molecular abnormalities outside of classical driver mutations. Notably, between 15-20% of patients will progress towards a leukemic transformation

This is a complex concern, Halpern says, as there is still uncertainty regarding the ability of current therapeutic strategies to alter disease trajectory. Accordingly, addressing these unmet needs is crucial for improving patient outcomes and enhancing their well-being in clinical practice, Halpern concludes.