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

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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.

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Authors and Affiliations

  1. Hematology, Department of Translational and Precision Medicine, Az. Policlinico Umberto I-Sapienza University, Via Benevento 6, 00161, Rome, Italy

    Sara Pepe, Emilia Scalzulli & Massimo Breccia

  2. Section of Hematology, Department of Radiological and Hematological Sciences, Catholic University, Fondazione Policlinico A. Gemelli IRCCS, Rome, Italy

    Elena Rossi & Valerio De Stefano

  3. Ospedale S. Eugenio, Rome, Italy

    Malgorzata Trawinska

  4. Hematology, Department of Clinical and Molecular Medicine, University Hospital Sant’Andrea, Sapienza University of Rome, Rome, Italy

    Caterina Tatarelli & Agostino Tafuri

  5. Belcolle Hospital, Viterbo, Italy

    Ambra Di Veroli & Roberto Latagliata

  6. Tor Vergata University, Rome, Italy

    Luca Maurillo

  7. Hematology, IFO, Rome, Italy

    Atelda Romano

  8. S. Giovanni Addolorata Hospital, Rome, Italy

    Sabrina Leonetti Crescenzi

  9. UOSD Ematologia ASL, Roma 1, Rome, Italy

    Tommaso Caravita di Toritto

  10. Spaziani Hospital, Frosinone, Italy

    Alessandro Andriani

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  1. Sara Pepe
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Contributions

All authors contributed to the study conception and design. The first draft of the manuscript was written by MB and SP; all authors commented on previous versions of the manuscript. All authors read and approved the final manuscript.

Corresponding author

Correspondence to Massimo Breccia.

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All procedures performed in studies involving human participants were in accordance with the ethical standards of the responsible institution and/or national research committee and with the 1964 Helsinki Declaration and its later amendments or comparable ethical standards. Informed consent was obtained from all patients for being included in the study.

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Pepe, S., Rossi, E., Trawinska, M. et al. Safety and effectiveness of ruxolitinib in the real-world management of polycythemia vera patients: a collaborative retrospective study by pH-negative MPN latial group. Ann Hematol 101, 1275–1282 (2022). https://doi.org/10.1007/s00277-022-04815-w

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