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Longitudinal Assessment of Transfusion Intensity in Patients With JAK Inhibitor-Naive or -Experienced Myelofibrosis Treated With Momelotinib Harrison, Claire mesa, ruben Talpaz, Moshe Gerds, Aaron Perkins, Andrew Gupta, Vikas Fox, Maria Laura Institut Català de la Salut [Harrison CN] Guy’s and St Thomas’ NHS Foundation Trust, London, UK. [Mesa R] Atrium Health Wake Forest Baptist Comprehensive Cancer Center, Winston-Salem, NC. [Talpaz M] University of Michigan Comprehensive Cancer Center, Ann Arbor, MI. [Gupta V] University of Toronto, Toronto, Ontario, Canada. [Gerds AT] Cleveland Clinic Taussig Cancer Center, Cleveland, OH. [Perkins A] Alfred Health and Monash University, Melbourne, VIC, Australia. [Fox ML] Vall d’Hebron Hospital Universitari, Barcelona, Spain. Experimental Hematology, Vall d’Hebron Institute of Oncology (VHIO), Barcelona, Spain Vall d'Hebron Barcelona Hospital Campus Sang - Transfusió Mielofibrosi - Tractament Proteïnes quinases - Inhibidors - Ús terapèutic Avaluació de resultats (Assistència sanitària) ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Therapeutics::Biological Therapy::Blood Transfusion CHEMICALS AND DRUGS::Chemical Actions and Uses::Pharmacologic Actions::Molecular Mechanisms of Pharmacological Action::Enzyme Inhibitors::Protein Kinase Inhibitors::Janus Kinase Inhibitors Other subheadings::Other subheadings::/therapeutic use DISEASES::Hemic and Lymphatic Diseases::Hematologic Diseases::Bone Marrow Diseases::Myeloproliferative Disorders::Primary Myelofibrosis Other subheadings::Other subheadings::Other subheadings::/drug therapy ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Diagnosis::Prognosis::Treatment Outcome TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::terapéutica::terapia biológica::transfusión sanguínea COMPUESTOS QUÍMICOS Y DROGAS::acciones y usos químicos::acciones farmacológicas::mecanismos moleculares de acción farmacológica::inhibidores enzimáticos::inhibidores de proteínas cinasas::inhibidores de las cinasas Janus Otros calificadores::Otros calificadores::/uso terapéutico ENFERMEDADES::enfermedades hematológicas y linfáticas::enfermedades hematológicas::enfermedades de la médula ósea::trastornos mieloproliferativos::mielofibrosis primaria Otros calificadores::Otros calificadores::Otros calificadores::/farmacoterapia TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::diagnóstico::pronóstico::resultado del tratamiento Anemia; Myeloproliferative neoplasm; Red blood cell transfusion Anemia; Neoplasia mieloproliferativa; Transfusión de glóbulos rojos Anèmia; Neoplàsia mieloproliferativa; Transfusió de glòbuls vermells 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. 2025-03-06T13:24:13Z 2025-03-06T13:24:13Z 2024 2025-03 info:eu-repo/semantics/article info:eu-repo/semantics/publishedVersion Harrison CN, Mesa R, Talpaz M, Gupta V, Gerds AT, Perkins A, et al. Longitudinal Assessment of Transfusion Intensity in Patients With JAK Inhibitor-Naive or -Experienced Myelofibrosis Treated With Momelotinib. Clin Lymphoma, Myeloma Leuk. 2025 Mar;25(3):199–211. 2152-2650 http://hdl.handle.net/11351/12700 10.1016/j.clml.2024.10.001 39516087 001427360500001 http://hdl.handle.net/11351/12700 eng Clinical Lymphoma, Myeloma and Leukemia;25(3) https://doi.org/10.1016/j.clml.2024.10.001 Attribution 4.0 International http://creativecommons.org/licenses/by/4.0/ info:eu-repo/semantics/openAccess application/pdf Elsevier Scientia