About this item:

49 Views | 23 Downloads

Author Notes:

John Mascarenhas, MD, Division of Hematology/Oncology, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, One Gustave L Levy Pl, Box 1079, New York, NY 10029, Email: john.mascarenhas@mssm.edu

AC received a 2018 ASH HONORS grant from the American Society of Hematology. JM receives research funding paid to the institution from Incyte, Roche, PharmaEssentia, Novartis, CTI Biopharma, Janssen, Promedior, Kartos, and Celgene; is on the clinical trial steering committee and an advisory board member for Incyte, Celgene, Roche, and Constellation Pharmaceuticals. JG has received research funding paid to the institution from Incyte, Novartis, CTI Biopharma, Janssen, Promedior, Kartos, and Celgene; is on the clinical trial steering committee and/or an advisory board member for Incyte, Novartis, Celgene, and Kartos. RR has received consulting fees from Constellation, Incyte, Celgene, Promedior, CTI, Jazz Pharmaceuticals, Blueprint, Stemline; and research funding from Incyte, Constellation, and Stemline. ATG has received research funding paid to the institution from Incyte, Roche, CTI Biopharma, Imago Biosciences, and Celgene; is on the clinical trial steering committee and an advisory board member for CTI Biopharma, Pfizer, Parmassentia, and Apexx Oncology. GSH has received research support from Bayer, Merck, Incyte and Constellation; is on the scientific advisory board for Incyte, BMS, Celgene, Agios, and Jazz; has received grants from K12 CA087723 Paul Calabresi Award, ASH-AMFDP, and Sanchez-Ferguson Award. EFW has received research funding from Incyte Corporation, Blueprint Medicine, and Sierra Oncology for clinical trials. The remaining authors have stated that they have no conflicts of interest.

Subject:

Keywords:

  • Science & Technology
  • Life Sciences & Biomedicine
  • Oncology
  • Hematology
  • Myeloproliferative
  • Neoplasm
  • Phlebotomy
  • RESPONSE
  • Splenomegaly
  • QUALITY-OF-LIFE
  • ESSENTIAL THROMBOCYTHEMIA
  • AVAILABLE THERAPY
  • DOUBLE-BLIND
  • SURVIVAL
  • EFFICACY
  • SAFETY
  • TRANSFORMATION
  • HYDROXYUREA
  • INTOLERANT

Real-World Outcomes of Ruxolitinib Treatment for Polycythemia Vera

Show all authors Show less authors

Tools:

Journal Title:

CLINICAL LYMPHOMA MYELOMA & LEUKEMIA

Volume:

Volume 20, Number 10

Publisher:

, Pages 697-+

Type of Work:

Article | Post-print: After Peer Review

Abstract:

Introduction: Ruxolitinib is approved for the treatment of polycythemia vera (PV) with hydroxyurea resistance or intolerance. Approval was based on the phase III RESPONSE trial, which demonstrated efficacy in a highly selected patient population. Materials and Methods: To characterize the tolerability and outcomes of ruxolitinib outside of a clinical trial, we performed a multi-center retrospective analysis of patients with PV treated with ruxolitinib at 11 participating sites across the United States. Outcomes of interest included change in phlebotomy requirements after starting ruxolitinib and spleen response, as these were included in the primary composite outcome in the RESPONSE trial. Results: One hundred twenty-six patients met eligibility criteria, and the median duration of follow-up was 22.4 months (range, 0-63.0 months). At 32 weeks after starting ruxolitinib, the percentage of patients who received at least 1 phlebotomy was significantly decreased compared with before ruxolitinib (37% vs. 56%; relative risk [RR], 0.66; 95% confidence interval [CI], 0.52-0.84; P <.001). Phlebotomy requirements were similarly decreased in patients who had received at least 3 phlebotomies prior to ruxolitinib initiation (28% vs. 17%; RR, 1.65; 95% CI, 1.13-2.40; P <.01). Resolution of palpable splenomegaly was also documented (48% vs. 20%; RR, 2.45; 95% CI, 1.70-3.53; P <.0001). A total of 9.5% of patients discontinued ruxolitinib owing to treatment-emergent adverse events, and 81.7% of patients were receiving ruxolitinib at last known follow-up. Conclusion: These real-world results are similar to those reported from the RESPONSE trial, although additional follow-up is necessary to assess long-term outcomes and potential for late-onset toxicity.

Copyright information:

This is an Open Access work distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (https://creativecommons.org/licenses/by-nc-nd/4.0/rdf).
Export to EndNote