Observational Study on the Use of Ropeginterferon Alfa-2b in Polycythemia Vera (ROPEG-PV)
NCT Number: NCT06506084
Status: RECRUITING
Study Type: OBSERVATIONAL
Conditions: Polycythemia Vera
Interventions: DRUG: Ropeginterferon alfa-2b
Sponsor: FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 319
Study Design: Observational Model: |Time Perspective: p
Start Date: 2022-09-08
Completion Date: 2026-12-31
Locations: UOC Ematologia, ASST Papa Giovanni XXIII, Bergamo, Lombardia, 24127, Italy|Divisione Ematologia ASST, Grande Ospedale Metropolitano Niguarda, Milano, Lombardia, 20162, Italy|Divisione Ematologia, Fondazione IRCCS Policlinico San Matteo, Pavia, Lombardia, 27100, Italy|U.O. Ematologia, Ospedale di Circolo e Fondazione Macchi Varese, Varese, Lombardia, 21100, Italy|Clinica Medica I Azienda Ospedaliera di Padova, Padova, Veneto, 35128, Italy|Divisione Ematologia, Ospedale Borgo Roma, Verona, Veneto, 37134, Italy|Divisione Ematologia, Ospedale San Bortolo, Vicenza, Veneto, Italy|A.S.O. SS. Antonio e Biagio e C.Arrigo di Alessandria, Alessandria, 15121, Italy|Azienda Ospedaliera Universitaria Consorziale - Policlinico, U.O. Ematologia con Trapianto, Bari, Italy|Policlinico S. Orsola - Malpighi, Unità di Ematologia, Bologna, Italy|ASST-Spedali Civili, Brescia, 25123, Italy|Ospedale Businco, S.C. Ematologia e CTMO, Cagliari, Italy|Azienda Ospedaliero - Universitaria "Policlinico Vittorio Emanuele" - PO Gaspare Rodolico, Dipartimento di Ematologia con Trapianto di midollo Osseo, Catania, Italy|Azienda Ospedaliera S. Croce e Carle di Cuneo- Divisione di Ematologia,, Cuneo, 2100, Italy|Arcispedale Sant'Anna Azienda Ospedaliero - Universitaria di Ferrara, Unità Operativa di Ematologia, Ferrara, Italy|Azienda Ospedaliera Universitaria Careggi, Divisione di Ematologia, Firenze, Italy|Azienda Ospedaliera Universitaria Policlinico "G. Martino", UOC Ematologia, Messina, Italy|Ospedale dell'Angelo, Dipartimento di Ematologia, Mestre, Italy|Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Ematologia, Milano, Italy|Ospedale San Raffaele, Unità Operativa di Ematologia e Trapianto Midollo Osseo, Milano, Italy|ASST MONZA Ospedale San Gerardo Clinica Ematologica, Monza, 20900, Italy|Azienda Ospedaliera Universitaria Federico II di Napoli Divisione di Ematologia e Trapianti del Midollo, Napoli, 80131, Italy|Azienda Ospedaliero Universitaria Maggiore della Carità di Novara SCDU Ematologia, Novara, 28100, Italy|Azienda Ospedaliera Universitaria Policlinico "P. Giaccone", Divisione di Ematologia, Palermo, Italy|Azienda Ospedaliera Universitaria Pisana, Pisa, Italy|Azienda Ospedaliera San Eugenio - UOC Ematologia, Roma, Italy|Fondazione Policlinico Universitario A. Gemelli - Università Cattolica del Sacro Cuore, UCSC Ematologia, Roma, Italy|Policlinico Umberto I, Dipartimento Ematologia, Oncologia e Dermatologia, Roma, Italy|Ospedale Casa Sollievo della Sofferenza Istituto di Ricovero e Cura a Carattere Scientifico, U.O. Ematologia, San Giovanni Rotondo, Italy|A.O.U. Città della Salute e della Scienza di Torino - Ospedale Molinette- S.C. Ematologia U, Torino, 10126, Italy|Ospedale Maggiore, SC Ematologia, Trieste, Italy|Azienda Sanitaria Universitaria Integrata, Presidio Ospedaliero "Santa Maria della Misericordia", Clinica Ematologica, Udine, Italy

Summary:
Polycythaemia vera (PV) is associated with a reduced quality of life, a high rate of vascular events, and an intrinsic risk of disease evolution. The results of several randomised trials for the treatment with new cytoreductive agents are now available, among which a new ropegylated formulation of interferon alfa-2b (ropeginterferon alfa-2b) have been recently approved in Europe and USA \[EMA (2019), FDA (2021) and AIFA (2022)\]. The use of this drug in clinical practice is an opportunity for a prospective observational study in a rare disease such as PV; the aim is to evaluate its impact in the practical management of these patients.

Therefore, the main objectives of the present study are to determine:

(i) to what extent ropeginterferon alfa-2b can be prescribed and tolerated in patients with PV; (ii) the risk-benefit of ropeginterferon alfa-2b in patients with PV, followed-up in real-world clinical practice.

Primary Outcome Measures: Complete hematological remission (CHR)* after 1 and 2 years of treatment., \*HCT lower than 45% without phlebotomies in the past 3 months; platelet count ≤ 400x109/L, WBC count \<10 x109/L, At baseline and during the follow up at 6/12/18/24 months per patient.
Secondary Outcome Measures: Screening failure, Percentage of patients who, after careful screening as required in good clinical practice, will fail the indications for concomitant clinical or laboratory abnormalities., At baseline and during the follow up at 6/12/18/24 months per patient.|Complete hematological (CHR) and clinical remission (CR) after 1 and 2 years of treatment stratified by different eligibility categories for treatment indication., Secondary Outcomes for efficacy, At baseline and during the follow up at 6/12/18/24 months per patient.|Dose-response effect on CHR and CR, Secondary Outcomes for efficacy, At baseline and during the follow up at 6/12/18/24 months per patient.|Frequency of phlebotomies, Secondary Outcomes for efficacy, At baseline and during the follow up at 6/12/18/24 months per patient.|Change in spleen size, Change in spleen size (from baseline) evaluated by palpation., At baseline and during the follow up at 6/12/18/24 months per patient.|Incidence of any of the following: o arterial and venous thrombotic events; o hemorrhagic events; o disease related symptoms; o disease evolutions into myelofibrosis and acute leukemia; o secondary malignancies., Secondary Outcomes for efficacy, At baseline and during the follow up at 6/12/18/24 months per patient.|Incidence, causality and intensity of any adverse event occurring during study period (as defined by MedDRA code and graded according to Common Terminology Criteria for Adverse Events (CTCAE last version))., Secondary Outcomes for safety, At baseline and during the follow up at 6/12/18/24 months per patient.