Clinical outcomes of interferon therapy for polycythemia vera and essential thrombocythemia: a systematic review and meta-analysis.

Interferon therapy has been used in clinical practice for more than three decades to treat polycythemia vera (PV) and essential thrombocythemia (ET). However, there has been no systematic investigation of its expected outcomes and potential risks. We performed a systematic review and single-arm meta-analysis to assess the clinical outcomes (hematological response, molecular response, vascular events, hematological transformation, and adverse events) after interferon therapy for patients with PV and ET. A systematic search identified 37 reports, including data from 1794 patients that were published before March 2021. The pooled overall hematological response (OHR) rate was 86%, with better OHR rates observed in studies using long-acting interferon (p < 0.001) and studies with younger patients (p = 0.038). The pooled overall molecular response rate was 48%, and inter-study heterogeneity was also related to patient age (p = 0.009). The overall incidence was 0.42/100 person-years for thrombosis, 0.01/100 person-years for hemorrhage, 0.21/100 person-years for myelofibrotic transformation, and 0.08/100 person-years for leukemic transformation. Compared with hydroxyurea, interferon produced a non-inferior hematological response and a superior molecular response. In conclusion, interferon therapy provided high rates of hematological and molecular response for patients with PV and ET and was associated with a favorable prognosis.