Long-Term Ropeginterferon Alfa-2b Is Safe, Effective in Polycythemia Vera

New data support the use of ropeginterferon alfa-2b therapy in patients with low-risk or high-risk polycythemia vera (PV), according to a presentation at the 2022 American Society of Hematology Annual Meeting.

Low-risk patients in particular achieved good hematologic and molecular responses with very few adverse events, and the analysis demonstrated benefits of longer-term treatment. The researchers, led by Jean-Jacques Kiladjian, MD, of the Center of Clinical Investigations at Hôpital Saint-Louis in Paris, France, reported that their data reinforces recently updated guidelines from European LeukemiaNet, which encourage early initiation of this treatment.

“Patients with ‘low-risk’ PV are managed conservatively despite higher risk of vascular events and impaired quality of life compared to a population without PV,” wrote the authors.

However, studies have found that ropeginterferon alfa-2b is an effective cytoreductive treatment with long-term efficacy and safety, as compared with hydroxyurea/best available treatment. The current research analyzed previous results in ropeginterferon alfa-2b subgroups to elucidate differences between risk categories.

The CONTINUATION-PV study randomized 46 patients with low-risk PV and 49 with high-risk disease to receive either ropeginterferon alfa-2b or hydroxyurea/best available treatment for a treatment duration of six years or longer. Low risk was defined as aged younger than 60 years and no history of thrombosis.

Both low-risk and high-risk patients received comparable doses throughout the course of the study. Low-risk patients achieved a higher complete hematologic response rate than high-risk patients over long-term treatment (by month 72, rates were 73.2% in the low-risk group versus 38.3% in the high-risk group). Low-risk patients also achieved molecular response more rapidly (median time to first response of 12 months versus 18 months). Regardless of risk level, most patients receiving ropeginterferon alfa-2b did not require phlebotomy in the sixth year of treatment.

The authors emphasized that in both the PROUD-PV and CONTINUATION-PV studies, only one low-risk patient (2% of the treatment cohort) withdrew due to an adverse drug reaction (sarcoidosis). In contrast, 13 (18.3%) high-risk patients withdrew due to events including thrombocytopenia, elevated transaminases, psychiatric disorder, autoimmune disorder, hypothyroidism, and dyspnea/pneumonitis.

Reference

Kiladjian J-J, Klade C, Georgiev P, et al. Efficacy and safety of long-term ropeginterferon alfa-2b treatment in patients with low-risk and high-risk polycythemia vera (PV). Abstract #4345. Presented at the 64th ASH Annual Meeting and Exposition; December 10-13, 2022; New Orleans, Louisiana.