Can pegylated interferon improve the outcome of polycythemia vera patients?

• Published in: Journal of hematology and oncology Vol. 10; no. 1; p. 15
• Main Authors: Crisà, Elena, Cerrano, Marco, Beggiato, Eloise, Benevolo, Giulia, Lanzarone, Giuseppe, Manzini, Paola Maria, Borchiellini, Alessandra, Riera, Ludovica, Boccadoro, Mario, Ferrero, Dario
• Format: Journal Article
• Language: English
• Published: England BioMed Central Ltd 13-01-2017; BioMed Central; BMC
• Subjects: Adolescent; Adult; Age; Aged; Care and treatment; Clinical trials; Disease Progression; Dosage and administration; Drug dosages; Female; Hematology; Humans; Hydroxyurea; Hydroxyurea - adverse effects; Hydroxyurea - therapeutic use; Interferon; Interferon-alpha - administration & dosage; Interferon-alpha - adverse effects; Interferon-alpha - therapeutic use; JAK2 allele burden; Janus Kinase 2 - genetics; Letter to the Editor; Male; Middle Aged; Neoplasms, Second Primary - chemically induced; Oncology; Patient outcomes; Patients; Pegylated interferon; Polycythemia; Polycythemia vera; Polycythemia Vera - complications; Polycythemia Vera - drug therapy; Polycythemia Vera - mortality; Polyethylene Glycols; Remission Induction; Risk factors; Survival Rate; Thrombosis; Thrombosis - chemically induced; Treatment Outcome; Young Adult; Hydroxyurea; JAK2 allele burden; Polycythemia vera; Pegylated interferon
• ISSN: 1756-8722; 1756-8722
• DOI: 10.1186/s13045-017-0395-1

Pegylated interferon (peg-IFN) was proven by phase II trials to be effective in polycythemia vera (PV); however, it is not clear whether it could improve patient outcome compared to hydroxyurea (HU). Here, we present an observational study on 65 PV patients aged 65 years or younger, who received either peg-IFN (30) or HU (35) according to the physician choice. Median follow-up was 75 months. The two cohorts were comparable for patient and disease characteristics. Eighty-seven percent of the patients treated with peg-INF responded, with a CR rate of 70% as compared to 100 and 49% with HU, respectively. Discontinuation rate was similar in the two groups (20% in peg-IFN vs 17% in HU). JAK2 allele burden was monitored in peg-INF arm only, and a reduction was observed in 88% of the patients. No thrombotic events were observed during peg-IFN treatment compared to three on HU. Disease progression to myelofibrosis or acute myeloid leukemia occurred to a patient only in peg-INF, compared to three in HU. Overall, three second malignancies were observed during the study, two in patients who received HU only, and one in a patient largely treated HU who received also peg-IFN for 3 months. Overall survival was significantly better for peg-IFN patients compared to HU, p = 0.027. Our study, albeit limited by small patient and event number and lack of randomization, confirms the efficacy of peg-INF in PV and shows a significant survival advantage for peg-INF-treated patients. Waiting for confirming data from the ongoing phase III trials, our study can support peg-INF as a first-line treatment option for PV, at least for younger patients.