Evaluation of Real-World Data on Ropeginterferon Alfa-2b in Patients With Polycythemia Vera: Insights From a Multicenter Study (MIRROR)

Multicenter Insights on Real-World Results Of Ropeginterferon Alpha-2B in Polycythemia Vera Patients

This retrospective study aims to evaluate the effectiveness and safety of Ropeginterferon Alfa-2b (BESREMI) in patients with Polycythemia Vera (PV). Eligible patients have a confirmed PV diagnosis according to current criteria, have received at least one dose of Ropeginterferon, and have complete clinical and laboratory data available. The primary objective is to analyze the time course of hematologic response (complete or partial, CHR/PR) according to ELN criteria, and to identify clinical and treatment-related factors associated with achieving and maintaining response. Secondary objectives include time to response, duration of response, progression-free survival, thromboembolic event rate, safety and tolerability, treatment discontinuation, dose modifications and adherence, normalization of hematologic parameters, and changes in JAK2 V617F allele burden. Data will be collected retrospectively from medical records at participating centers.

Study Overview

• Status: Not yet recruiting
• Conditions: Polycytemia Vera
• Intervention / Treatment: Drug: Ropeginterferon alfa-2b (BESREMi®)

• Study Type: Observational
• Enrollment (Estimated): 150

Contacts and Locations

• Study Contact: Name: Novella Pugliese, MD, PhD; Phone Number: +393396137232; Email: novypugliese@yahoo.it

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The study will include patients diagnosed with Polycythemia Vera (PV) according to current diagnostic criteria who have received at least one administration of Ropeginterferon Alfa-2b (BESREMI) as part of their disease management. Eligible patients must have complete clinical and laboratory information available and must be followed at one of the participating centers. All participants must provide written informed consent prior to inclusion in the study.

Description

Inclusion Criteria:

• Confirmed diagnosis of Polycythemia Vera according to updated diagnostic criteria
• Have received at least one dose of Ropeginterferon Alfa-2b (BESREMI).
• Complete clinical and laboratory data availability for review (e.g., hematologic parameters, treatment response, adverse events).
• Followed at one of the participating study centers.
• Have signed informed consent for participation.

Exclusion Criteria:

• Have received experimental or non-approved treatments for PV during the observation period.
• Lack sufficient clinical or laboratory data to allow inclusion in the analysis.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Patients with Polycythemia Vera Receiving Ropeginterferon aplha 2b; Data will be collected retrospectively from patient medical records and will include: Demographic Data; Clinical Characteristics of PV; Treatment Information. Details on Ropeginterferon Alfa-2b therapy: Dosage; Treatment schedule and duration; Clinical and hematologic response; Treatment discontinuation and reasons for interruption; Clinical and Hematologic Response: Hematologic parameters (hemoglobin, hematocrit, platelet count, etc.); Clinical status during treatment; Spleen size assessed via abdominal ultrasound (when available); Documentation of baseline values, prior therapies, dosing schedules, and response assessments at: 3, 6, 9, 12, 18, and 24 months; Discontinuation rates and reasons for stopping therapy; Molecular Response: Quantification of JAK2 V617F allele burden over time; Adverse Events: Any clinically relevant complications or side effects

Drug: Ropeginterferon alfa-2b (BESREMi®); Ropeginterferon Alfa-2b was administered in accordance with the approved prescribing information.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Longitudinal Analysis of Hematologic Response (CR/PR) to Ropeginterferon Alfa-2b in polycythemia vera	Definition: Proportion of patients achieving Complete Hematologic Response (CHR) or Partial Response (PR) at each scheduled timepoint (3, 6, 9, 12, 18 and 24 months), classified according to the ELN criteria and identification of baseline and on-treatment factors associated with (a) first attainment of CHR/PR and (b) maintenance (duration) of CHR/PR. Assessment timepoints: Baseline, 3, 6, 9, 12, 18 and 24 months (or last available follow-up). Summary metrics: Proportion (%) in CHR and PR at each timepoint	From enrollment to the end of treatment or at least 12 months of treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to Hematologic Response	Time (in months) from the first day of Ropeginterferon Alfa-2b treatment to achievement of CHR or PR hematologic response according to ELN criteria.	Baseline to first documented CHR or PR (up to 24 months)
Duration of Hematologic Response	Interval between first documented CHR or PR and loss of that response, death, or last follow-up.	From first CHR/PR to loss or last follow-up (up to 24 months)
Progression-Free Survival (PFS)	Time from treatment initiation to progression to post-PV myelofibrosis, acute myeloid leukemia, or death from any cause	Baseline to event or last follow-up (up to 24 months)
Thromboembolic Event Rate	Proportion of patients experiencing arterial or venous thrombotic or embolic events during treatment with Ropeginterferon.	Baseline to last follow-up (up to 24 months)
Incidence of Treatment-Emergent Adverse Events	Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]) CTCAE v6.	Baseline to last follow-up (up to 24 months)
Treatment Discontinuation	Proportion of patients who discontinue Ropeginterferon for adverse events, lack of efficacy, or other clinical reasons.	Baseline to last follow-up (up to 24 months)
Dose Modifications and Adherence	Number and proportion of patients with significant dose changes (reductions or increases) and estimated adherence (planned vs observed dosing intervals).	Baseline to last follow-up (up to 24 months)
Normalization of Hematologic Parameters	Proportion of patients achieving target hematologic values: hematocrit <45%, leukocytes <10000/mm³, platelets <400000/mm³.	Baseline and at scheduled visits (3,6,9,12,18,24 months)
JAK2 V617F Allele Burden	Assessment of JAK2 V617F allele burden dynamics during Ropeginterferon treatment and correlation with hematologic response.	Baseline and at scheduled visits (3,6,9,12,18,24 months)

Collaborators and Investigators

• Sponsor: Federico II University

Study record dates

Study Major Dates

• Study Start (Estimated): January 10, 2026
• Primary Completion (Estimated): March 26, 2026
• Study Completion (Estimated): March 31, 2026

Study Registration Dates

• First Submitted: November 26, 2025
• First Submitted That Met QC Criteria: December 11, 2025
• First Posted (Actual): December 15, 2025

Study Record Updates

• Last Update Posted (Actual): December 15, 2025
• Last Update Submitted That Met QC Criteria: December 11, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: Ropeginterferon alpha 2b
• Other Study ID Numbers: AIFA-RSO-3948 (Other Identifier: A.O.U. Federico II Napoli)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Individual Participant Data (IPD) will not be shared to protect participant privacy and to comply with ethical and regulatory requirements.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No