Event‐free survival in early polycythemia vera patients correlates with molecular response to ropeginterferon alfa‐2b or hydroxyurea/best available therapy (PROUD‐PV/CONTINUATION‐PV)

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Clonal expansion of JAK2V617F-mutated hematopoietic stem cells underlies almost all cases of PV, a rare myeloproliferative neoplasm.1 Nevertheless, the clinical relevance of reducing the variant allele frequency (VAF) (i.e., achieving molecular response [MR] to treatment) has been disputed.Prospective trials in PV are challenging given the latent onset and low penetrance of major outcomes such as thrombotic events and progression to myelofibrosis or acute myeloid leukemia. 2Therefore, recent analyses demonstrating a correlation of MR with prolonged EFS in high-risk PV patients resistant/intolerant to hydroxyurea generated strong interest.3,4 If confirmed in a wider PV population, these findings suggest MR could be a major treatment aim.We examined the relationship between MR and EFS in the PROUD-PV/CONTINUATION-PV studies (#NCT01949805; #NCT02218047) in an early-stage PV population and explored the clinical relevance of further depleting the JAK2V617F clone.The phase 3 randomized PROUD-PV study and its extension CONTINUATION-PV enrolled low-and high-risk PV patients requiring cytoreduction who were treatment-naïve or hydroxyurea pretreated for ≤3 years without resistance/intolerance.Design and methods have been published previously.5,6 Patients were randomized 1:1 to receive ropeginterferon alfa-2b or hydroxyurea for 12 months.Ropeginterferon alfa-2b (BESREMi®) was started at a dose of 50-100 μg every 2 weeks and escalated until blood counts normalized, with a maximum dose of 500 μg.Hydroxyurea was administered at 500-3000 mg daily.Patients who enrolled in CONTINUATION-PV remained in their allocated treatment arm.Control arm patients could switch from hydroxyurea to any standard HemaSphere.

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