Safety and effectiveness of ruxolitinib in the real-world management of polycythemia vera patients: a collaborative retrospective study by pH-negative MPN latial group.

Abstract

Ruxolitinib is approved for polycythemia vera (PV) patients after failure to previous cytoreductive therapy, based on durable results observed in phase 3 trials. We report a multicenter retrospective study demonstrating the efficacy and safety of ruxolitinib in real-life setting. Eighty-three patients were evaluated. Median follow-up was 24.5 months (IQR 14.0-29.3). At a 3-month response assessment, ruxolitinib provided significant benefit in reducing hematocrit (HCT) level (p < 0.001), phlebotomy requirement (p < 0.001), leucocytes (p = 0.044), and disease-related symptoms (p < 0.001). The exposure-adjusted rates (per 100 patient-years) of infectious complications, thromboembolic events, and secondary malignancies were 6.9, 3, and 3.7, respectively. Non-melanoma skin cancers (NMSC) were the most frequent (40%) SM type. Lymphoproliferative disorders were not detected. Five (6%) patients permanently discontinued ruxolitinib treatment and four (5%) evolved in myelofibrosis (MF), but none in acute leukemia. The rate of MF evolution per 100 patient-years of exposure was 2.8. In our experience, ruxolitinib confirmed its efficacy and safety outside of clinical trials.

• Authors: Sara Pepe, Elena Rossi, Malgorzata Trawinska, Caterina Tatarelli, Ambra Di Veroli, Luca Maurillo, Atelda Romano, Sabrina Leonetti Crescenzi, Tommaso Caravita di Toritto, Agostino Tafuri, Roberto Latagliata, Emilia Scalzulli, Alessandro Andriani, Valerio De Stefano, Massimo Breccia
• Journal: Annals of hematology (Ann Hematol) Vol. 101 Issue 6 Pg. 1275-1282 (Jun 2022) ISSN: 1432-0584 [Electronic] Germany
• PMID: 35318505 (Publication Type: Journal Article, Multicenter Study)
• Copyright: © 2022. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany, part of Springer Nature.

Chemical References

• Nitriles
• Pyrazoles
• Pyrimidines
• ruxolitinib

Topics

• Humans
• Hydrogen-Ion Concentration
• Nitriles (therapeutic use)
• Polycythemia Vera (complications)
• Primary Myelofibrosis (diagnosis)
• Pyrazoles (adverse effects)
• Pyrimidines
• Retrospective Studies