Dr. Mascarenhas on Approved and Emerging Treatment Options in Myelofibrosis


John O. Mascarenhas, MD, discusses approved and emerging treatment options in myelofibrosis.

John O. Mascarenhas, MD, associate professor of medicine, hematology, and medical oncology at the Icahn School of Medicine at Mount Sinai; director of the Adult Leukemia Program; and leader of Clinical Investigation within the Myeloproliferative Disorders Program at Mount Sinai; and a member of the Tisch Cancer Institute, discusses approved and emerging treatment options in myelofibrosis.

The field of myelofibrosis is becoming more competitive as alternative treatments for patients who progress on ruxolitinib (Jakafi) enter the pipeline, explains Mascarenhas.

One agent, fedratinib (Inrebic), received FDA approval for the treatment of patients with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis, says Mascarenhas.

The JAKARTA-2 study, results of which led to the approval of fedratinib, evaluated the agent in the second-line setting. The agent led to significant reduction in splenomegaly in patients who progressed on ruxolitinib, Mascarenhas explains. Although the agent is associated with some gastrointestinal toxicities, it provides another JAK inhibitor option in this space.

Other novel agents such as CPI-0610 are being evaluated in combination with ruxolitinib in patients who had a suboptimal response to ruxolitinib, says Mascarenhas. Additionally, the BCL-2 inhibitor navitoclax is being explored in the myelofibrosis space.

Ultimately, the goal of clinical trials is to develop novel therapies to better manage spleen symptoms, improve quality of life, and increase overall survival rates, concludes Mascarenhas.

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