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Clinical Trial
. 2025 Mar;25(3):199-211.
doi: 10.1016/j.clml.2024.10.001. Epub 2024 Oct 16.

Longitudinal Assessment of Transfusion Intensity in Patients With JAK Inhibitor-Naive or -Experienced Myelofibrosis Treated With Momelotinib

Claire N Harrison  1 , Ruben Mesa  2 , Moshe Talpaz  3 , Vikas Gupta  4 , Aaron T Gerds  5 , Andrew Perkins  6 , Yeow Tee Goh  7 , Maria Laura Fox  8 , Donal McLornan  9 , Jeanne Palmer  10 , Lynda Foltz  11 , Alessandro Vannucchi  12 , Steffen Koschmieder  13 , Francesco Passamonti  14 , Sung-Eun Lee  15 , Catherine Ellis  16 , Bryan Strouse  16 , Francisco J Gonzalez Carreras  17 , Stephen T Oh  18
Affiliations
Free article
Clinical Trial

Longitudinal Assessment of Transfusion Intensity in Patients With JAK Inhibitor-Naive or -Experienced Myelofibrosis Treated With Momelotinib

Claire N Harrison et al. Clin Lymphoma Myeloma Leuk. 2025 Mar.
Free article

Abstract

Purpose: Anemia is a cardinal feature of myelofibrosis often managed with red blood cell (RBC) transfusions, which may contribute to negative prognostic, quality-of-life, and healthcare-related economic impacts. The Janus kinase (JAK) 1/JAK2/activin A receptor type 1 inhibitor momelotinib was approved for the treatment of patients with myelofibrosis and anemia based on clinical trial evidence of anemia, spleen, and symptom benefits illustrated using binomial response/nonresponse endpoints. In the present post hoc, descriptive analyses, the impact of momelotinib on RBC transfusion burden over time was further characterized across JAK inhibitor-naive and -experienced patients.

Methods: All RBC units transfused were collected during the baseline and 24-week treatment periods, initially in a single-arm phase 2 study as proof-of-concept analysis, and then versus comparators (ruxolitinib, best available therapy [BAT], and danazol) in the phase 3 SIMPLIFY-1, SIMPLIFY-2, and MOMENTUM studies, respectively.

Results: In the phase 2 study, mean transfusion requirement changed by -1.5 units/28 days, with 85% of patients (35/41) achieving numeric transfusion reduction. Across SIMPLIFY-1, SIMPLIFY-2, and MOMENTUM, mean transfusion requirements decreased with momelotinib (-0.1, -0.36, and -0.86 units/28 days), while mean requirements with ruxolitinib, BAT, and danazol changed by +0.39, 0, and ‒0.28 units/28 days, respectively. Overall, 87% (185/213), 77% (79/103), and 85% (110/130) of patients had improved or stable transfusion intensities with momelotinib versus 54% (117/216), 62% (32/52), and 63% (41/65) with ruxolitinib, BAT, and danazol.

Conclusion: These novel time-dependent transfusion burden analyses demonstrate that momelotinib is associated with anemia-related benefits in most patients and greater transfusion burden reduction versus comparators.

Trial registration: ClinicalTrials.gov identifiers: NCT02515630, NCT01969838, NCT02101268, NCT04173494.

Keywords: Anemia; Hemoglobin; Janus kinase inhibitor; Myeloproliferative neoplasm; Red blood cell transfusion.

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Conflict of interest statement

Disclosure CNH has received institutional research funding from BMS/Celgene, Constellation Pharmaceuticals Inc (a MorphoSys Company), and Novartis; consulting fees from AOP, Galecto, GSK, Keros, SOBI, and Roche; advisory role and speaker funding from AbbVie, AOP Pharma, BMS/Celgene, Constellation Pharmaceuticals Inc (a MorphoSys Company), CTI BioPharma, Galecto, Geron, GSK, Jannsen, Novartis, Roche, and Promedior; and support from Novartis for attending meetings. RM reports consulting fees from AbbVie, Blueprint, BMS, CTI Biopharma, Genentech, Geron, GSK, Incyte, MorphoSys, Novartis, and Sierra Oncology. MT reports research funding from BMS and advisory board participation for BMS, Kyowa Kirin, and SDP/Sumitomo. VG reports consulting fees from AbbVie, BMS Celgene, Daiichi Sankyo, GSK, and Pfizer and participation in a data safety monitoring/advisory board for AbbVie, BMS Celgene, and Daiichi Sankyo. ATG reports consulting fees from AbbVie, BMS, Constellation/MorphoSys, CTI Biopharma, Imago Biosciences/Merck, Incyte, PharmaEssentia, Sierra Oncology, and Telios. AP reports payment of honoraria from and participation in advisory board work with AbbVie, CTI BioPharma, GSK, Incyte, Kartos, and Novartis. YTG reports consulting fees from Amgen, Antengene, Astellas, AstraZeneca, GSK, Janssen, Pfizer, and Roche; and payment or honoraria from AbbVie, DKSH, and Recordati. MLF reports consulting fees from AbbVie, GSK, Novartis, Sanofi, and Sierra Oncology and participation in a Keros Therapeutics steering committee. DM reports grants from Imago Biosciences; payment or honoraria from AbbVie, Jazz Pharmaceuticals, and Novartis; participation in a data safety monitoring board for the UK ALL RIC trial; and a leadership role with the European Society for Blood and Marrow Transplantation. JP reports payment or honoraria from CTI Biopharma and participation in a data safety monitoring/advisory board for MorphoSys. LF reports honoraria from GSK and Novartis and advisory board participation with GSK, Medison, and Novartis. AV reports payment or honoraria from AbbVie, AOP, BMS, GSK, Incyte, Novartis, and Roche. SK reports research funding, consulting fees, honoraria, and/or travel grants from AbbVie, AOP Pharma, BMS/Celgene, CTI Biopharma, Geron, GSK, Incyte, Imago Biosciences, iOMEDICO, Janssen, Kartos, MPN Hub, MSD, Novartis, Pfizer, PharmaEssentia, and Sierra Oncology and intellectual property with RWTH Aachen University. FP reports grants from BMS; consulting fees from AbbVie, AOP Health, BMS/Celgene, Karo Pharma, Kyowa Kirin, MEI, Novartis, Roche, Sierra Oncology, and Sumitomo; and payment or honoraria from AbbVie, AOP Health, BMS/Celgene, Novartis, and Roche. STO reports consulting fees from AbbVie, Constellation, BMS, Cogent, CTI Biopharma, Geron, Incyte, Protagonist, and Sierra Oncology. CE, BS, and FJGC report employment with and stock/stock options at GSK. SEL declares no competing interests.

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