Polycytemia Vera
Conditions
Keywords
Ropeginterferon alpha 2b
Brief summary
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.
Interventions
Ropeginterferon Alfa-2b was administered in accordance with the approved prescribing information.
Sponsors
Federico II University
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Longitudinal Analysis of Hematologic Response (CR/PR) to Ropeginterferon Alfa-2b in polycythemia vera | From enrollment to the end of treatment or at least 12 months of treatment | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Duration of Hematologic Response | From first CHR/PR to loss or last follow-up (up to 24 months) | Interval between first documented CHR or PR and loss of that response, death, or last follow-up. |
| Progression-Free Survival (PFS) | Baseline to event or last follow-up (up to 24 months) | Time from treatment initiation to progression to post-PV myelofibrosis, acute myeloid leukemia, or death from any cause |
| Thromboembolic Event Rate | Baseline to last follow-up (up to 24 months) | Proportion of patients experiencing arterial or venous thrombotic or embolic events during treatment with Ropeginterferon. |
| Incidence of Treatment-Emergent Adverse Events | Baseline to last follow-up (up to 24 months) | Incidence of Treatment-Emergent Adverse Events \[Safety and Tolerability\]) CTCAE v6. |
| Time to Hematologic Response | Baseline to first documented CHR or PR (up to 24 months) | Time (in months) from the first day of Ropeginterferon Alfa-2b treatment to achievement of CHR or PR hematologic response according to ELN criteria. |
| Dose Modifications and Adherence | Baseline to last follow-up (up to 24 months) | Number and proportion of patients with significant dose changes (reductions or increases) and estimated adherence (planned vs observed dosing intervals). |
| Normalization of Hematologic Parameters | Baseline and at scheduled visits (3,6,9,12,18,24 months) | Proportion of patients achieving target hematologic values: hematocrit \<45%, leukocytes \<10000/mm³, platelets \<400000/mm³. |
| JAK2 V617F Allele Burden | Baseline and at scheduled visits (3,6,9,12,18,24 months) | Assessment of JAK2 V617F allele burden dynamics during Ropeginterferon treatment and correlation with hematologic response. |
| Treatment Discontinuation | Baseline to last follow-up (up to 24 months) | Proportion of patients who discontinue Ropeginterferon for adverse events, lack of efficacy, or other clinical reasons. |
Contacts
Primary ContactNovella Pugliese, MD, PhD
Outcome results
None listed