Abstracts Clinical Lymphoma, Myeloma & Leukemia September 2023 S402 Sponsorship: Incyte Corporation Keywords: MPN, myelofibrosis, Janus kinase, anemia, hepcidin, Phase I MPN-549 Transfusion‑Related Cost and Time Burden Offsets in Patients With Myelofibrosis Treated With Momelotinib vs Best Available Therapy Lucia Masarova MD1, Tom Liu MS2, Sumati Rao PhD2, Mirko Fillbrunn PhD3, Weilong Li PhD4, Gautam Sajeev ScD3, Boris Gorsch Pharm.D2, James Signorovitch PhD3 1The University of Texas MD Anderson Cancer, Houston, USA. 2GSK plc, Philadelphia, USA. 3Analysis Group, Inc., Boston, USA. 4Analysis Group, Inc., New York, USA Context: Transfusion-dependent anemia is present in onequarter of patients with myelofibrosis at diagnosis and becomes more common over time. Transfusion dependence (TD) imposes a high cost burden, with annual total medical costs up to 9× higher than non-TD. SIMPLIFY-2 was a randomized Phase III trial of the JAK1/JAK2/ACVR1 inhibitor momelotinib vs best available therapy (BAT; 88.5% ruxolitinib) in ruxolitinib-experienced patients with myelofibrosis; momelotinib was associated with higher red blood cell (RBC) transfusion independence (TI) rates vs BAT. Objective: To estimate projected differences between momelotinib and BAT in total medical costs and outpatient transfusion visit cost/time burdens, including in patients aged ≥65 years. Design: Analyses were based on mean outpatient transfusion visits and transfusion status over 24 weeks in SIMPLIFY-2 (TD: ≥4 RBC units transfused or a hemoglobin <8 g/dL in the prior 8 weeks; TI: no RBC transfusions and all hemoglobin ≥8 g/dL in the prior 12 weeks; transfusion requiring [TR]: neither TD nor TI). Projected cost offsets were calculated using TD vs TI/TR cost estimates from the IBM MarketScan database and, for patients aged ≥65 years, the Medicare Fee-for-Service database. Projected time savings were based on transfusion rates and previous time burden estimates. Results: Projected average annual total medical cost savings based on week 24 transfusion status with momelotinib vs BAT were $47,484/patient with baseline TD ($19,526/patient ≥65 years) and $15,226/patient with baseline TI/TR (−$42/patient ≥65 years due to similar week 24 TD rates for momelotinib and BAT among this subpopulation). Fewer outpatient transfusion visits with momelotinib vs BAT resulted in projected annual savings of $10,143/patient with baseline TD ($12,269/patient ≥65 years) and $5,857/patient with baseline TI/TR ($5,427/patient ≥65 years). Among patients with baseline TD, projected annual time savings in transfusion visits for momelotinib vs BAT totaled 42 hours/patient (16 hours preparation/waiting, 20 hours transfusion/recovery, 6 hours travel), and 51 hours/patient ≥65 years. Conclusions: Reductions in TD and transfusion visits for momelotinib vs BAT resulted in projected healthcare system savings in transfusion costs and transfusion-related time burden in ruxolitinib-experienced patients with myelofibrosis, including patients aged ≥65 years. Keywords: MPN, myelofibrosis, momelotinib, transfusion, anemia, Phase III MPN-551 Clinical Outcomes With Momelotinib vs Ruxolitinib in Patients With Myelofibrosis and Anemia: Subgroup Analysis of SIMPLIFY‑1 Vikas Gupta MD1, Stephen Oh MD, PhD2, Timothy Devos MD, PhD3, Viviane Dubruille MD4, John Catalano MD5, Timothy Somervaille MD, PhD6, Pilar Giraldo MD, PhD7, Hiroshi Kosugi MD8, Tomasz Sacha MD, PhD9, Jiri Mayer MD, PhD10, Arpad Illes MD, PhD11, Catherine Ellis BA12, Zhaohui Wang MS12, Jun Kawashima MD13, Ruben Mesa MD14 1Princess Margaret Cancer Centre, Toronto, Canada. 2Washington University School of Medicine, St Louis, USA. 3University Hospitals Leuven and KU Leuven, Leuven, Belgium. 4CHU de Nantes, Nantes, France. 5Monash University & Frankston Hospital, Frankston, Australia. 6The Christie NHS Foundation Trust & Cancer Research UK Manchester Institute, Manchester, United Kingdom. 7Miguel Servet University Hospital and Centro de Investigacion Biomedica en Red de Enfermadades Raras, Zaragoza, Spain. 8Ogaki Municipal Hospital, Ogaki, Japan. 9Jagiellonian University Hospital, Kraków, Poland. 10University Hospital Brno, Brno, Czech Republic. 11Division of Haematology, Department of Internal Medicine, Faculty of Medicine, University of Debrecen, Debrecen, Hungary. 12GSK plc, Philadelphia, USA. 13Sierra Oncology, a GSK company, San Mateo, USA. 14Wake Forest University School of Medicine, Winston-Salem, USA Context: The JAK1/JAK2/ACVR1 inhibitor momelotinib has shown clinical benefit in addressing symptoms, spleen volume, and anemia across 3 phase III studies in patients with myelofibrosis: SIMPLIFY-1 (NCT01969838), SIMPLIFY-2 (NCT02101268), and MOMENTUM (NCT04173494). In MOMENTUM, all patients were anemic at baseline (hemoglobin <10 g/dL) and previously treated with a JAK inhibitor. In contrast, SIMPLIFY-1 enrolled JAK inhibitor–naïve patients and included those with and without anemia, but outcomes in anemic subgroups have not been comprehensively reported. Objective: To descriptively characterize the efficacy and safety of momelotinib vs ruxolitinib in anemic patient subgroups from SIMPLIFY-1. Design: In SIMPLIFY-1, JAK inhibitor–naïve patients with intermediate-1–, intermediate-2–, or high-risk myelofibrosis were randomized 1:1 to momelotinib or ruxolitinib. There were no hemoglobin requirements for enrollment; anemic subgroups are defined here by baseline hemoglobin <10 or <12 g/dL. Week 24 endpoints included the rates of spleen volume reduction ≥35% (SVR35), Total Symptom Score (TSS) response (≥50% reduction), and transfusion independence response (TI-R; no red blood cell transfusions or hemoglobin <8 g/dL for terminal 12 weeks). Results: Of 432 patients, 42% and 75% had baseline hemoglobin <10 or <12 g/dL, respectively; the proportion of patients in these anemic subgroups was similar between arms. In these subgroups, SVR35 rates were generally consistent with those observed in the intent-to-treat (ITT) population (momelotinib, 27%; ruxolitinib, 30%), at 31% vs 33% (<10 g/dL) and 29% vs 29% (<12 g/dL). TSS response rates in these anemic subgroups were also generally consistent with the ITT (momelotinib, 28%;
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