title: Can pegylated interferon improve the outcome of polycythemia vera patients?
abstract: 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.
authors.affiliation: Hematology Division, Università degli Studi di Torino, Via Genova 3, 10126, Turin, Italy.
authors.forename: Elena
authors.initials: E
authors.lastname: Crisà
authors.affiliation: Hematology Division, Università degli Studi di Torino, Via Genova 3, 10126, Turin, Italy. cerranomarco@gmail.com.
authors.forename: Marco
authors.identifier: 0000-0003-1666-3100
authors.initials: M
authors.lastname: Cerrano
authors.affiliation: Hematology Division, Università degli Studi di Torino, Via Genova 3, 10126, Turin, Italy.
authors.forename: Eloise
authors.initials: E
authors.lastname: Beggiato
authors.affiliation: S.C. Hematology, A.O. Città della Salute e della Scienza, Turin, Italy.
authors.forename: Giulia
authors.initials: G
authors.lastname: Benevolo
authors.affiliation: Hematology Division, Università degli Studi di Torino, Via Genova 3, 10126, Turin, Italy.
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Lanzarone
authors.affiliation: Transfusion Medicine Unit, A.O. Città della Salute e della Scienza, Turin, Italy.
authors.forename: Paola Maria
authors.initials: PM
authors.lastname: Manzini
authors.affiliation: Hematology Division, Università degli Studi di Torino, Via Genova 3, 10126, Turin, Italy.
authors.forename: Alessandra
authors.initials: A
authors.lastname: Borchiellini
authors.affiliation: Section of Pathology, Department of Molecular Biotechnology and Health Sciences, University of Torino, Turin, Italy.
authors.forename: Ludovica
authors.initials: L
authors.lastname: Riera
authors.affiliation: Hematology Division, Università degli Studi di Torino, Via Genova 3, 10126, Turin, Italy.
authors.forename: Mario
authors.initials: M
authors.lastname: Boccadoro
authors.affiliation: Hematology Division, Università degli Studi di Torino, Via Genova 3, 10126, Turin, Italy.
authors.forename: Dario
authors.initials: D
authors.lastname: Ferrero
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D018450:Disease Progression; D005260:Female; D006801:Humans; D006918:Hydroxyurea / Q000009:adverse effects / Q000627:therapeutic use; D016898:Interferon-alpha / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use; D053614:Janus Kinase 2 / Q000235:genetics; D008297:Male; D008875:Middle Aged; D016609:Neoplasms, Second Primary / Q000139:chemically induced; D011087:Polycythemia Vera / Q000150:complications / Q000188:drug therapy* / Q000401:mortality; D011092:Polyethylene Glycols; D012074:Remission Induction; D015996:Survival Rate; D013927:Thrombosis / Q000139:chemically induced; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D016898:Interferon-alpha; D011092:Polyethylene Glycols; C507924:JAK2 protein, human; D053614:Janus Kinase 2; D006918:Hydroxyurea
keywords: Hydroxyurea; JAK2 allele burden; Pegylated interferon; Polycythemia vera