Dr Desai on the Treatment Landscape of Polycythemia Vera

“One of the biggest challenges [in treating patients with polycythemia vera] has been [elucidating] how to modify the underlying pathobiology of the disease.”

Ruchi J. Desai, MD, an assistant professor in the Division of Hematology, Oncology, and Palliative Care at the Virginia Commonwealth University (VCU) School of Medicine as well as a hematologist/oncologist in the Department of Internal Medicine at VCU Health, discussed the present treatment landscape of polycythemia vera (PV) and the remaining unmet needs in the space.

Desai began by noting that PV has historically been a disease which has led to significant symptom morbidity for patients. PV also carries risks for thrombosis, with a greater risk for arterial thrombosis compared with venous thrombosis, she added. Thus, treatment approaches have generally been focused on reducing this symptom morbidity, mainly through keeping hematocrit levels low via approaches such as phlebotomy and/or cytoreductive agents, she explained.

One of the biggest ongoing challenges in treating patients with PV has been determining how to modify the underlying pathobiology of the disease, Desai said. There has never been a true proven treatment approach that has been able to achieve this disease modification, she noted. The closest agent that has been able to modify the disease biology of PV is ropeginterferon alfa-2b-njft (Besremi), she explained.

Clinical studies of ropeginterferon alfa demonstrated a reduction in the JAK2 allele burden, which may be correlated to patients achieving a molecular and pathologic remission, Desai explained. However, what constitutes pathologic and molecular remissions in PV is not well defined, representing an unmet need in the field, she concluded.

Ropeginterferon alfa-2b was approved by the FDA for the treatment of patients with PV in November 2021. The regulatory decision was supported by data from the phase 3 PROUD-PV (NCT01949805) and CONTINUATION-PV (NCT02218047) studies which demonstrated that patients who received ropeginterferon alfaachieved a complete hematological response at a rate of 61% after 7.5 years of therapy.