Hematologic and molecular response to ropeginterferon alfa-2b in patients with polycythemia vera: a systematic review and meta-analysis
Clinical Snapshot
PICO Framework
| P — Population | Patients with polycythemia vera |
| I — Intervention | Ropeginterferon alfa-2b treatment |
| C — Comparator | No specific comparator mentioned in abstract (appears to be single-arm studies) |
| O — Outcomes | Complete hematologic response (CHR) and JAK2V617F variant allele frequency (VAF) reduction over 36 months |
Bottom Line
This meta-analysis suggests ropeginterferon alfa-2b achieves progressive hematologic and molecular responses in polycythemia vera patients over 36 months, with complete hematologic response rates increasing from 19% to 73%. However, the evidence quality appears limited by significant heterogeneity between studies and very wide confidence intervals, particularly the 17-97% range for 36-month response rates. The substantial variability in early responses raises questions about patient selection and treatment protocols. While the molecular response data showing progressive JAK2V617F reduction is encouraging, the absence of safety data and comparison with standard treatments like hydroxyurea limits clinical decision-making. Australian clinicians should await more robust evidence before considering this as first-line therapy, though it may represent a valuable option for selected patients in specialist hematology practice.
Key Findings
P Value: Not reported in abstract
Effect Size: Progressive increase in CHR from 19% at 3 months to 73% at 36 months
Primary Outcome: Complete hematologic response (CHR) at 36 months: 73% (95% CI: 17-97%)
Nnt Or Sensitivity: JAK2V617F VAF reduction of -54.90% (95% CI: -65.61 to -43.99%) at 36 months
Confidence Interval: 95% CI: 0.17-0.97 for CHR at 36 months
Clinical Application
Requires specialist hematology care and regular monitoring for molecular responses Would require TGA approval and PBS listing consideration. Fits within RACGP guidelines for specialist referral of myeloproliferative neoplasms Patients with polycythemia vera suitable for interferon therapy
Abstract
Ropeginterferon alfa-2b is an interferon used in the treatment of myeloproliferative neoplasms, particularly polycythemia vera. Its efficacy in achieving hematologic and molecular responses has been demonstrated in clinical trials, but pooled data on long-term outcomes and sustained response remain limited. This systematic review and meta-analysis aimed to evaluate the hematologic and molecular response over 36 months. PubMed, Scopus, Science Direct, and Google Scholar databases were searched to identify studies reporting hematologic and molecular responses to ropeginterferon alfa-2b. Studies were included if they provided data on complete hematologic response (CHR) and JAK2V617F variant allele frequency (VAF) reduction. Pooled proportions and mean reductions were calculated using random-effects models. The pooled proportion of CHR increased progressively from 0.19 (95% CI: 0.04-0.57) at 3 months to 0.73 (95% CI: 0.17-0.97) at 36 months. Molecular response, measured by VAF reduction, deepened over time from - 7.33 (95% CI: -9.85 to -4.81) at 3 months to -54.90 (95% CI: -65.61 to -43.99) at 36 months. Subgroup analyses revealed significant variability in response rates, particularly in early follow-up periods. Ropeginterferon alfa-2b achieves significant and sustained hematologic and molecular responses over 36 months. This makes it a promising treatment for polycythemia vera. While variability in early responses needs further investigation, the sustained long-term efficacy compared to hydroxyurea supports its use in clinical practice. Future studies should focus on identifying predictors of response and optimizing treatment protocols to maximize patient outcomes.
References
- 1.Elgadi, A., Wagealla, M., Noorallah, T., Esmail, R., & Almahi, S. S. (2026). Hematologic and molecular response to ropeginterferon alfa-2b in patients with polycythemia vera: a systematic review and meta-analysis. Annals of Hematology. https://doi.org/10.1038/sj.leu.2404854
Related Research
Blood advances
Phase 1b study of ABBV-744, a novel, selective BET inhibitor, as monotherapy for patients with myelofibrosis
27 May 2026
European journal of pediatrics
Safety and efficacy of liposomal iron for iron deficiency anemia in pediatric population: A systematic review and meta-analysis of randomized controlled trials
26 May 2026
The Cochrane database of systematic reviews
Early erythropoiesis-stimulating agents in preterm or low-birthweight infants
25 May 2026
This content is for educational purposes for healthcare professionals only and does not constitute clinical advice. Clinical decisions should be based on individual patient assessment, current guidelines, and appropriate specialist consultation. Editorial Standards · Privacy Policy · Terms of Service