Dr. Verstovsek on Limitations of the Current Treatment Landscape of Myelofibrosis

Srdan Verstovsek, MD, PhD, United Energy Resources, Inc. Professor of Medicine, director of the Hanns A. Pielenz Clinical Research Center for Myeloproliferative Neoplasms, and chief, Section for Myeloproliferative Neoplasms, Department of Leukemia, The University of Texas MD Anderson Cancer Center, discusses the limitations of the current treatment landscape of myelofibrosis.

Myelofibrosis is an aggressive myeloproliferative neoplasm that is associated with limited survival, says Verstovsek. Currently, the JAK inhibitors ruxolitinib (Jakafi) and fedratinib (Inrebic) represent the only approved treatment options in myelofibrosis.

Treatment with ruxolitinib can improve symptom burden, decrease spleen size, and improve quality of life (QOL), explains Verstovsek. Moreover, patients tend to gain weight, be more active, and have improved metabolism and organ function with ruxolitinib.

Fedratinib is a similar agent to ruxolitinib and may have utility as a second-line option for patients who progress on ruxolitinib, says Verstovsek.

While up to 75% of patients should be candidates for JAK inhibitors, treatment is based on how symptomatic patients are and their QOL, says Verstovsek. In reality, 30% to 40% of patients are exposed to JAK inhibition.

Additionally, anemia remains a clinical unmet need in myelofibrosis, adds Verstovsek. As the treatment options are limited, many patients require off-label use of medications to manage anemia, boost potential JAK inhibitor response, or be used as treatment after progression on ruxolitinib or fedratinib.

In addition to JAK inhibitors, transplant offers a potentially curative option for patients; however, less than 10% of patients with myelofibrosis undergo transplant, concludes Verstovsek.