@article{1ad9dca49a4a4b359f8e294784955b9d,
title = "Real-World Outcomes of Ruxolitinib Treatment for Polycythemia Vera",
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.",
keywords = "Myeloproliferative, Neoplasm, Phlebotomy, RESPONSE, Splenomegaly",
author = "Alexander Coltoff and Ruben Mesa and Jason Gotlib and Jessica Shulman and Rampal, {Raajit K.} and Olivia Siwoski and Abdulraheem Yacoub and Alison Moliterno and Anna Yang and Evan Braunstein and Gerds, {Aaron T.} and Hobbs, {Gabriela S.} and Winton, {Elliott F.} and Swati Goel and Martha Wadleigh and Douglas Tremblay and Erin Moshier and John Mascarenhas",
note = "Funding Information: 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. Funding Information: 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. Publisher Copyright: {\textcopyright} 2020 Elsevier Inc.",
year = "2020",
month = oct,
doi = "10.1016/j.clml.2020.05.019",
language = "English (US)",
volume = "20",
pages = "697--703.e1",
journal = "Clinical Lymphoma, Myeloma and Leukemia",
issn = "2152-2650",
publisher = "Cancer Media Group",
number = "10",
}