# Real-World Retrospective Report on the Efficacy, Tolerability, and Molecular Responses to Ropeginterferon-α2b in Patients with Myeloproliferative Neoplasms

**Authors:** Matthias Christen, Domenic Kaderli, Milos Ratknic, Adrián Dante de Angelis, Philipp Stefan Aebi, Naomi Porret, Joëlle Tchinda, Natalia Baran, Wuddri Rim, Pascale Julia Tanner, Sebastian Mathes, Anne Angelillo-Scherrer, Alicia Rovó, Sara C. Meyer

PMC · DOI: 10.3390/jcm15010128 · Journal of Clinical Medicine · 2025-12-24

## TL;DR

This study examines how well Ropeginterferon-α2b works in treating myeloproliferative neoplasms in real-world settings, showing improved blood control and manageable side effects.

## Contribution

The paper provides real-world evidence of Ropeginterferon-α2b's efficacy and tolerability across multiple MPN subtypes with molecular monitoring.

## Key findings

- Hematologic control improved from 45% at treatment start to 60% at last follow-up.
- JAK2V617F variant allele frequency decreased in patients with serial monitoring.
- Ropeginterferon-α2b was generally well tolerated, though 15% discontinued due to side effects.

## Abstract

Background: Ropeginterferon alfa-2b (Ropeg-IFNa) is increasingly used in myeloproliferative neoplasms (MPN), particularly polycythemia vera, but real-world data across subtypes are limited. We evaluated clinical and molecular responses to Ropeg-IFNa in routine practice. Methods: We retrospectively analyzed 20 JAK2V617F-positive MPN patients treated at a tertiary center. Baseline features, dosing, treatment line, hematologic responses, adverse events, and serial JAK2V617F variant allele frequency (VAF) were extracted from records. Results: Median age at initiation was 53 years; 55% were ELN high-risk. Ropeg-IFNa was started first-line or after peginterferon alfa-2a, hydroxyurea, or a tapered JAK2 inhibitor. Mean treatment duration was 14 ± 11 months at 195 ± 143 µg Q2W. Hematologic control increased from 45% at the start to 60% at the last follow-up. Among patients with serial molecular monitoring (n = 11), median JAK2V617F VAF declined from 21.2 to 12.7%. Ropeg-IFNa was generally well tolerated; adverse effects were mostly manageable, although 3/20 (15%) discontinued due to side effects, including mood disturbances, while others continued with supportive care and dose adjustments. Conclusions: In this single-center cohort, Ropeg-IFNa was tolerable and associated with improved hematologic control and modest VAF reductions, supporting its use in multi-subtype MPN cohorts. These findings underscore the value of longitudinal driver-mutation monitoring during therapy.

## Linked entities

- **Genes:** JAK2 (Janus kinase 2) [NCBI Gene 3717]
- **Diseases:** myeloproliferative neoplasms (MONDO:0020076), polycythemia vera (MONDO:0009891)

## Full-text entities

- **Genes:** JAK2 (Janus kinase 2) [NCBI Gene 3717] {aka JTK10}
- **Diseases:** mood disturbances (MESH:D019964), MPN (MESH:D009369), polycythemia vera (MESH:D011087)
- **Chemicals:** hydroxyurea (MESH:D006918), Ropeginterferon-alpha2b (-)
- **Species:** Homo sapiens (human, species) [taxon 9606]
- **Mutations:** JAK2V617F

## Full text

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## Figures

4 figures with captions in the complete paper: https://tomesphere.com/paper/PMC12787081/full.md

## References

28 references — full list in the complete paper: https://tomesphere.com/paper/PMC12787081/full.md

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Source: https://tomesphere.com/paper/PMC12787081