Abstracts Clinical Lymphoma, Myeloma & Leukemia September 2023 S400 Outcome Measures: Percentage of patients with STD/LTD, number of workdays lost, and associated indirect/direct healthcare costs during 12-month follow-up. Results: A total of 10,711 patients were included (MF, n=173; PV, n=4477; ET, n=6061). The median age was 54 (MF), 51 (PV), and 48 years (ET). Total direct healthcare costs were significantly higher among all MPN cohorts vs. controls (all P<0.001). MF patients had the largest mean cost difference of $67,456 higher (ET $22,279; PV $10,970 higher). MPN cohorts had significantly higher percentages of patients with an STD claim, a larger number of STD-related workdays lost, and higher STDassociated indirect costs vs controls. Patients with MF had an 8.1% higher chance of filing an STD claim (ET 7.5%, PV 3.3% higher). Results were consistent but less pronounced for LTD. Compared with matched TE controls, PV and ET patients with TEs were more likely to have an inpatient stay and had higher all-cause total healthcare costs, STD-related workdays lost, and STD-related indirect costs (all P<0.05). Results were consistent but less pronounced for LTD. The small sample size precluded MF TE cohort analyses. Conclusions: Economic burden was high among employed MPN patients. Patients with MF, PV, and ET were significantly more likely to take disability leave and had higher direct/indirect costs vs matched controls. TEs significantly impacted healthcare costs, and STD leave for PV and ET patients vs controls. Sponsorship: Incyte. Keywords: MPN, polycythemia vera, myelofibrosis, essential thrombocythemia, thromboembolic events MPN-540 Disease Progression and Leukemic Transformation in Patients With Lower‑Risk Myelofibrosis: An Analysis From the Myelofibrosis and Essential Thrombocythemia Observational Study (MOST) Rami Komrokji MD1, Michael R. Grunwald MD2, Evan Braunstein MD PhD3, J.E. Hamer-Maansson MSPH3, Tricia Kalafut PhD3, John Mascarenhas MD4 1Malignant Hematology Department, H. Lee Moffitt Cancer Center and Research, Tampa, FL, USA. 2Department of Hematologic Oncology and Blood Disorders, Levine Cancer Institute, Atrium Health, Charlotte, NC, USA. 3Incyte Corporation, Wilmington, DE, USA. 4Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA Context: Progressive disease (PD) in myelofibrosis is associated with increased bone marrow fibrosis, worsening anemia, and increased circulating blasts, and can result in leukemic transformation (LT). Real-world data regarding PD and LT in lower-risk myelofibrosis are limited. Objective: An analysis of MOST (NCT02953704) describing PD/LT incidence in lower-risk patients and comparing characteristics of patients with vs without physician-reported PD. Design: Longitudinal, noninterventional, prospective study. Patients: Adults with DIPSS low/intermediate-1–risk myelofibrosis (age >65 years alone). Results: Of 232 patients enrolled, 204 were analyzed (cutoff: 09/30/2022; with PD, n=59; without PD, n=134; unknown, n=11; median [range] time from diagnosis to enrollment, 1.8 [0-38] years; median [range] enrollment duration, 4.4 [3.5-5.6] years). Versus non-PD patients, PD patients were older (median [range], 70.0 [35-88] vs 66.5 [35-88] years), more were Black (10.2% vs 5.2%), fewer were employed (20.3% vs 44.4%); and more were unable to work (16.9% vs 2.3%). Among PD patients, PD indicators were reported (per physician) as changes in hematologic parameters (45.8%), spleen size (32.2%), myelofibrosis symptoms (27.1%), and blasts (13.6%); 25.4% had LT; 20.3% died from PD. More low-risk patients had changes in blasts (20.0% vs 10.3%); fewer had LT (20.0% vs 28.2%); fewer died from PD (10.0% vs 25.6%) vs intermediate-1 risk. Symptomology of patients with vs without PD at enrollment was not significantly different. At enrollment, 117/204 patients (57.4%) received myelofibrosis-directed monotherapy (hydroxyurea, 44.4%; ruxolitinib, 41.9%). Of patients receiving monotherapy, more with PD received ruxolitinib (62.9% vs 32.4%) and fewer received hydroxyurea (25.7% vs 51.4%), vs without PD. However, more patients receiving ruxolitinib vs hydroxyurea at enrollment had intermediate-1–risk myelofibrosis (65.3% vs 48.1%), palpable spleen (50.0% vs 28.2%), and a longer median myelofibrosis duration from diagnosis to enrollment (2.6 vs 1.1 years) and from diagnosis to PD (4.3 vs 3.0 years). Conclusions: In this real-world analysis of MOST, approximately one-third of patients had physician-reported PD; these patients were older, less likely employed/able to work, more likely Black, and more likely receiving ruxolitinib vs hydroxyurea at enrollment. Patients receiving ruxolitinib more likely had signs of progression at enrollment, suggesting greater ruxolitinib use in higher PD-risk myelofibrosis. Keywords: myelofibrosis, leukemic transformation, progressive disease MPN-541 Targeted Therapy of Uncontrolled Erythrocytosis in Polycythemia Vera With the Hepcidin Mimetic, Rusfertide: Blinded Randomized Withdrawal Results of the Phase 2 Revive Study Naveen Pemmaraju MD1, Marina Kremyanskaya MD, PhD2, Andrew Kuykendall MD3, Ellen Ritchie MD4, Jason Gotlib MD5, Aaron Gerds MD6, Jeanne Palmer MD7, Kristen Pettit MD8, Uttam Nath MD9, Abdulraheem Yacoub MD10, Arturo Molina MD, MS11, Nishit Modi PhD11, Frank Valone MD11, Sarita Khanna PhD11, Suneel Gupta PhD11, Srdan Verstovsek MD, PhD1, Yelena Ginzburg MD2, Ronald Hoffman MD2 1MD Anderson Cancer Center, Houston, USA. 2Icahn School of Medicine at Mount Sinai Hospital, New York, USA. 3H.Lee Moffitt Cancer Center and Research Institute, Tampa, USA. 4Weill Cornell Medical College of Cornell University, New York, USA. 5Stanford Hospital, Palo Alto, USA. 6Cleveland Clinic Taussig Cancer Center, Cleveland, USA. 7Mayo Clinic Hospital, Phoenix, USA. 8University of Michigan, Ann Arbor, USA. 9All India Institute of Medical Sciences, Rishikesh, Rishikesh, India. 10The University Of Kansas Medical Center, Kansas City, USA. 11Protagonist Therapeutics, Inc, Newark, USA
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