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Observational Study
. 2024 Aug;103(8):2837-2843.
doi: 10.1007/s00277-024-05735-7. Epub 2024 Apr 25.

Hematocrit control and thrombotic risk in patients with polycythemia vera treated with ruxolitinib in clinical practice

Aleksander Chojecki  1 , Danielle Boselli  2 , Allison Dortilus  2 , Issam Hamadeh  3 , Stephanie Begley  2 , Tommy Chen  2 , Rupali Bose  2 , Nikolai Podoltsev  4 , Amer M Zeidan  4 , Nicole Baranda Balmaceda  5 , Abdulraheem Yacoub  5 , Jing Ai  6 , Thomas Gregory Knight  6 , Brittany Knick Ragon  6 , Nilay Arvind Shah  6 , Srinivasa Reddy Sanikommu  6 , James Symanowski  2 , Ruben Mesa  6 , Michael Richard Grunwald  6
Affiliations
Observational Study

Hematocrit control and thrombotic risk in patients with polycythemia vera treated with ruxolitinib in clinical practice

Aleksander Chojecki et al. Ann Hematol. 2024 Aug.

Abstract

Polycythemia vera (PV) is a myeloproliferative neoplasm characterized by unregulated red blood cell production resulting in elevated hemoglobin and/or hematocrit levels. Patients often have symptoms such as fatigue, pruritus, and painful splenomegaly, but are also at risk of thrombosis, both venous and arterial. Ruxolitinib, a selective Janus kinase inhibitor, is approved by the US Food and Drug Administration as second-line cytoreductive treatment after intolerance or inadequate response to hydroxyurea. Although ruxolitinib has been widely used in this setting, limited data exist in the literature on ruxolitinib treatment patterns and outcomes among patients with PV in routine clinical practice. We report a retrospective, observational, cohort study of patients treated for PV with ruxolitinib across three US centers (academic and regional practice) from December 2014-December 2019. The study included 69 patients, with a median follow-up duration of 3.7 years (95% CI, 2.9-4.4). Our data demonstrate very high rates of hematocrit control (88% of patients by three months and 89% by six months); few patients required dose adjustments or suspension. No arterial thromboses were observed; however, the follow-up duration does not allow for the generation of meaningful conclusions from this. Three patients had thrombotic events; one was in the setting of a second malignancy, one post-operative, and a third related to prolonged immobility. We also found that 28% of patients initiated ruxolitinib as a result of poorly controlled platelet counts, second only to hydroxyurea intolerance (46%) as a reason to start therapy. In clinical practice, ruxolitinib continues to be effective in controlling hematocrit levels after three and six months of treatment in patients and is associated with low thrombotic risk.

Keywords: Hematocrit; Myeloproliferative disorders; Polycythemia vera; Retrospective studies; Ruxolitinib; Thrombosis.

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Conflict of interest statement

The following relevant COI: Support for the present manuscript provided by Incyte as institutional support/ research funding, Grant number I-Rux-16-67Other disclosures directly or indirectly related to this work but reported by authors per Springer editorial policies include the following: Consulting Fees and Honoraria: Constellation Pharmaceuticals, AbbVie, Amgen, Astellas, Blueprint Medicines, Bristol Myers Squibb, Cardinal Health, CTI BioPharma, Daiichi Sankyo, Gamida Cell, Genentech, Gilead, GSK/Sierra Oncology, Incyte, Invitae, Jazz, Karius, Novartis, Ono Pharmaceutical, Pfizer, Pharmacosmos, Premier, Servier/Agios, and Stemline TherapeuticsPayment or honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events: Incyte, Research to Practice, Novartis, Sierra Oncology, Genentech, Sierra, Blueprint, Geron, Telios, CTI Biopharma, Incyte, BMS, Abbvie, Glaxo Smith Kline, Morphosys, Gilead, AstraZeneca, Cogen Biosciences, Abbvie, Janssen Oncology, Epizyme -honoraria or speakers bureau--Of note -- No writing support by Incyte for this manuscriptParticipation on a Data Safety Monitoring Board or Advisory Board: Novartis, Pfizer, Incyte, CTI, PharmaAstellas, Apellis, Gilead, Notable Labs, Celgene, AbbVie, Pfizer, Celgene/BMS, Jazz, Incyte, Agios, Servier, Boehringer-Ingelheim, Novartis, Astellas, Daiichi Sankyo, Geron, Taiho, Seattle Genetics, BeyondSpring, Takeda, Ionis, Amgen, Janssen, Roche, Genentech, Epizyme, Syndax, Gilead, Kura, Chiesi, ALX Oncology, BioCryst, Notable, Orum, Mendus, Zentalis, Schrodinger, Regeneron, Syros, Schrodinger, Tyme, Morphosys, Pharmaessentia, Acceleron Pharma, Epizyme, Adaptive Biotechnologies, ADC Therapeutics, Genmab, TG Therapuetics, Karyopharm, Loxo/Lilly Leadership or fiduciary role in other board, society, committee or advocacy group, paid or unpaid: Novartis, Abbvie, Gilead, Syros, BioCryst, Abbvie, ALX Oncology, Kura, Geron and Celgene/BMS, Leukemia Lymphoma SocietyStock or stock options: MedtronicOther financial or non-financial interests/Institutional Research Funding: Celgene/BMS, Abbvie, Astex, Pfizer, Kura, Medimmune/AstraZeneca, Boehringer-Ingelheim, Incyte, Takeda, Novartis, Shattuck Labs, Geron, Foran, Prelude, Incyte, Kartos, and Aprea.

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