Effective Management of Polycythemia Vera With Ropeginterferon Alfa-2b Treatment.

Area of Science:

• Hematology
• Oncology
• Pharmacology

Background:

• Polycythemia vera (PV) is a myeloproliferative neoplasm.
• Ropeginterferon alfa-2b is a novel, long-acting interferon for PV treatment.
• Standard dosing involves lower initial doses with gradual titration.

Purpose of the Study:

• To evaluate the efficacy and safety of a higher starting dose (250 µg) and simplified dose escalation of ropeginterferon alfa-2b in PV patients.
• To assess complete hematological response (CHR) rates and molecular response.
• To evaluate the safety profile of the new dosing regimen.

Main Methods:

• Forty-nine patients with PV and hydroxyurea intolerance were enrolled.
• Biweekly treatment with an initial dose of 250 µg, escalating to 350 µg and 500 µg.
• Efficacy assessed by CHR rates (European LeukemiaNet criteria), JAK2V617F allelic burden, and spleen size; safety monitored throughout.

Main Results:

• CHR rates reached 61.2% at week 24, 69.4% at week 36, and 71.4% at week 52.
• Significant reduction in JAK2V617F allelic burden from 58.5% to 30.1% by week 52.
• Durable responses, rapid time to CHR (median 5.6 months), spleen size reduction, and a favorable safety profile with mostly mild, reversible AEs.

Conclusions:

• The 250-350-500 µg dosing regimen of ropeginterferon alfa-2b is well-tolerated and effective for PV management.
• This regimen induces significant hematological and molecular responses and reduces spleen size.
• The findings support this simplified, higher-dose regimen as a viable treatment option for PV.