Dr. Rampal on Unmet Needs in Myelofibrosis and Polycythemia Vera

Raajit K. Rampal, MD, PhD
Published: Tuesday, Jan 14, 2020



Raajit K. Rampal, MD, PhD, hematologic oncologist, Memorial Sloan Kettering Cancer Center, discusses unmet needs in the treatment of patients with myelofibrosis and polycythemia vera.

In 2011, the JAK1/2 inhibitor ruxolitinib (Jakafi) became the first approved agent designed to treat patients with myelofibrosis. In 2014, ruxolitinib became the first drug to receive regulatory approval for the treatment of patients with polycythemia vera. In August 2019, fedratinib (Inrebic), a JAK2 inhibitor, was approved for the treatment of patients with intermediate-2 or high-risk primary or secondary myelofibrosis.

Beyond these agents, there are few options that are available to patients, explains Rampal. About half of patients with myelofibrosis who are treated with ruxolitinib will progress within 1.5 to 3 years. Similarly, response rates could decrease over time for patients with polycythemia vera who are treated with ruxolitinib.

Therefore, clinical trials should be considered for patients with either myelofibrosis or polycythemia vera who progress on these agents, concludes Rampal.
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Raajit K. Rampal, MD, PhD, hematologic oncologist, Memorial Sloan Kettering Cancer Center, discusses unmet needs in the treatment of patients with myelofibrosis and polycythemia vera.

In 2011, the JAK1/2 inhibitor ruxolitinib (Jakafi) became the first approved agent designed to treat patients with myelofibrosis. In 2014, ruxolitinib became the first drug to receive regulatory approval for the treatment of patients with polycythemia vera. In August 2019, fedratinib (Inrebic), a JAK2 inhibitor, was approved for the treatment of patients with intermediate-2 or high-risk primary or secondary myelofibrosis.

Beyond these agents, there are few options that are available to patients, explains Rampal. About half of patients with myelofibrosis who are treated with ruxolitinib will progress within 1.5 to 3 years. Similarly, response rates could decrease over time for patients with polycythemia vera who are treated with ruxolitinib.

Therefore, clinical trials should be considered for patients with either myelofibrosis or polycythemia vera who progress on these agents, concludes Rampal.



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