PEGylated Interferon-α Treatment in Polycythemia Vera and Essential Thrombocythemia: A Single Center, Real-Life Experience

Abstract

PEGylated interferon-alpha (Peg-IFN) is a highly effective therapy for polycythemia vera (PV) and essential thrombocythemia (ET), owing to its lower immunogenicity and reduced toxicity compared with conventional interferon-alpha. This retrospective study aimed to evaluate the efficacy on hematological response, the response generation process and safety of Peg-IFN alfa-2a treatment in patients with PV or ET. In this study, 68 patients with diagnosed ET or PV who had received Peg-IFN alfa-2a between May 2016 and May 2022 were enrolled. Peg-IFN therapy response assessment was performed according to the European Leukemia Net and the International Working Group-Myeloproliferative Neoplasms Research and Treatment consensus revised response criteria. Forty patients (58.8%) were diagnosed with ET and 28 (41.2%) with PV. In patients with PV and ET, ORR was 53.6%, 85.7%, 86.6% and 67.6%. 90.9%, 89.5% at the 3, 12, and 24 months, respectively. There was a significant effect of Peg-IFN on leukocyte counts and thrombocyte counts over time was observed in patients diagnosed with both PV and ET (p= p=0.001 vs p=0.026). In addition, a significant effect of time on hematocrit values was observed in PV patients (p=0.014). Adverse events were observed in 75.4% of the overall cohort, of which 72.9% were hematological and 96.8% were non-hematological adverse events. Peg-IFN is an effective treatment with high response rates and, despite its frequent adverse effects. The maintenance of treatment without premature discontinuation may play a crucial role in the observed high efficacy and significant hematological response.