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.
Patients diagnosed with Polycythemia Vera by WHO 2016
Patient aged ≥ 18 years old
Patients in need of cytoreductive treatments with ropeginterferon alfa-2b in first or later lines according to the reimbursability criteria defined by the Italian National Health System
Patients who have signed the written informed consent for study participation.
None
The first time point to assess the primary outcome defined as the achievement of CHR is after 12 months of treatment with ropeginterferon alfa-2b, is reasonable to provide evidence of at least a non-inferiority in terms of CHR with this new drug. In this framework, a sample size is calculated to test 1-sample non-inferiority of a proportion. Setting the expected proportion p of CHR under ropeginterferon alfa-2b equals to the reference value (p0) obtained with ruxolitinib (i.e., p = p0 = 0.24), a sample size of 319 is needed to demonstrate that the proportion of CHR under ropeginterferon alfa-2b is not inferior to that obtained under ruxolitinib, allowing a margin of non-inferiority (delta) of 0.07 and assuming alpha and beta errors of 5% and 10%, respectively.
Case-only
Prospective
Ropeginterferon alfa-2b
DrugData will be collected at each visit during the observational study (total duration: 24 months). In accordance with routine clinical practice in this patient population, visits are expected to take place every 6 months. Patients who discontinue ropeginterferon alfa-2b treatment at any time will no longer be followed up.
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
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.
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
Dose-response effect on CHR and CR
Secondary Outcomes for efficacy
Frequency of phlebotomies
Secondary Outcomes for efficacy
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