BESREMi® Outperforms in Reducing Symptomatic Burden of Polycythemia Vera: ASCO Real-World Analysis

A medical chart review published in the Journal of Clinical Oncology supplement has highlighted the efficacy of cytoreductive therapies (CTs) in reducing the burden of severe symptoms in patients with polycythemia vera (PV). The study, presented at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting, underscores the clinical utility of these therapies, especially ropeginterferon alfa-2b, marketed as BESREMi, which showed a significant reduction in severe PV symptoms.

The retrospective review involved 420 patient charts from 125 healthcare providers in the U.S., spanning from October to December 2023. Patients were divided into groups based on their treatment: phlebotomy alone (103 patients), and various CTs with or without phlebotomy, including hydroxyurea (165 patients), ruxolitinib (115 patients), peginterferon alfa-2a (44 patients), and ropeginterferon alfa-2b (45 patients). Observations revealed a notable decrease in severe symptoms across all CT groups. Particularly, ropeginterferon alfa-2b demonstrated the highest efficacy, reducing the number of patients experiencing severe symptoms by 75%, from 12 to 3 patients. Hydroxyurea, ruxolitinib, and peginterferon alfa-2a also showed reductions, though to a lesser extent.

The review also explored the rationale behind the initiation of cytoreductive therapy among patients initially treated with phlebotomy alone. The primary reasons cited by physicians were worsening symptoms (32%), increased frequency of phlebotomy sessions (29%), and difficulty in controlling hematocrit levels (24%). These insights emphasize the importance of symptom management in PV treatment beyond mere hematologic response.

Adan Sosa, Director of Health Economics and Outcomes Research at PharmaEssentia, noted the significance of these findings in enhancing clinical practice. He stressed that, given PV's chronic and potentially life-threatening nature, effective symptom management is crucial. The inclusion of BESREMi in the updated 2023 National Comprehensive Cancer Network Clinical Practice Guidelines for both high- and low-risk PV patients further validates its clinical utility.

PharmaEssentia's Head of Medical, Robert B. Geller, emphasized that real-world data reinforce the importance of addressing patient symptoms in treatment decisions. He expressed hope that the findings would encourage early and optimal use of cytoreductive interventions like BESREMi, which has shown meaningful clinical benefits for PV patients.

Polycythemia vera is a chronic blood disorder originating from a diseased stem cell in the bone marrow, leading to excessive production of red and white blood cells and platelets. This condition can cause serious cardiovascular complications, such as thrombosis and embolism, and may progress to secondary myelofibrosis or leukemia. Although the precise molecular mechanisms behind PV are still under investigation, acquired mutations, particularly in the JAK2 gene, play a crucial role.

BESREMi, developed by PharmaEssentia, is a monopegylated, long-acting interferon designed to be administered biweekly or monthly, depending on patient stability. Approved by regulatory bodies in the U.S., Europe, Taiwan, and South Korea, BESREMi has become a preferred therapeutic option for adult PV patients. However, it carries a boxed warning for serious disorders, including neuropsychiatric, autoimmune, ischemic, and infectious conditions, necessitating close monitoring and possible discontinuation in severe cases.

PharmaEssentia, headquartered in Taipei, Taiwan, continues to innovate in hematology, oncology, and immunology. Founded in 2003, the company leverages its scientific expertise to develop biologics for challenging diseases, with a growing global presence and a world-class production facility in Taichung, Taiwan.

The findings from this review highlight the critical role of cytoreductive therapies in managing PV symptoms and support the continued use and exploration of BESREMi in clinical practice. As treatments evolve, the focus on patient symptoms and comprehensive care remains paramount in improving outcomes for those with PV.