Burden of Anemia in Myelofibrosis Patients in Japan, South Korea, Taiwan, and Canada: A Real-World Survey and Multinational Database Analysis

Introduction Myelofibrosis (MF) is a myeloproliferative neoplasm characterized as a clonal stem cell-disorder. The current standard of care for MF includes Janus kinase inhibitors (JAKi); however, despite alleviating splenomegaly and symptoms, some JAKi treatments do not modify the underlying disease and can worsen anemia and thrombocytopenia. About 40% of patients (pts) with MF have moderate to severe anemia at diagnosis. Some JAKi treatments may induce or exacerbate MF-related anemia, which is a significant limitation. Anemia and transfusion requirements are associated with poor outcomes and the burden is underrecognized in some parts of the world. The study reported here explored physicians' perspectives on the management of pts with MF and anemia, and examined chart reviews to determine pt characteristics. Methods Adelphi MF I Disease Specific Programme™ data were derived from a cross-sectional survey with retrospective data collection comprising hematologists and hemato-oncologists in Japan, South Korea, Taiwan, and Canada, with data collection estimated to be between May and October 2024. Data on physicians' perceptions on treatment management and disease burden were collected within the physician survey, with pt level chart data captured within patient record forms (PRFs). PRFs were completed by physicians for their next 3-4 consecutively consulting pts, with each physician completing 1 PRF for pts with low-risk (LR) or intermediate-risk (IR) MF and 3 PRFs for pts with high-risk (HR) MF (per the International Prognostic Scoring System [IPSS] risk classification system). This abstract presents interim data (cutoff: July 2024) and the full dataset will be presented in the poster (publication funded by GSK [221309]). Results At interim analysis, surveys were completed by 26 physicians (Japan: n=5, South Korea: n=1, Taiwan: n=10, Canada: n=10), who managed a median (range) of 10.5 (2-100) pts with MF. For LR, IR, and HR pts, physicians reported transfusion dependence (TD; defined as pts receiving ≥6 units of red blood cell/whole blood transfusions in 12 weeks or 4 units in 8 weeks) in a median (range) of 10% (0-50%; n=25), 30% (0-100%; n=25), and 60% (0-100%; n=26), respectively. Out of all physicians (n=26), most considered reducing the need for transfusions (88 [LR]-92% [IR and HR]), improving or stabilizing Hb levels (80 [LR]-92% [HR]), and reducing fatigue (76 [LR]-88% [HR]) as either very or extremely important when choosing treatment regimens for pts across all risk levels. Across the IPSS risk levels (LR: n=25; IR: n=26; HR: n=25), the most important factors for determining the most appropriate treatment and management plan for pts with MF were pt quality of life (QoL; LR: 36% [9/25]; IR: 31% [8/26]; HR: 20% [5/25]), risk classification (LR: 20% [5/25]; IR: 27% [7/26]; HR: 28% [7/25]), Hb levels (LR: 16% [4/25]; IR: 12% [3/26]; HR: 4% [1/25]), and pt preferences/requests (LR: 16% [4/25]; IR: 4% [1/26]; HR: 12% [3/25]). Regardless of IPSS score, maintaining QoL (LR: 76%, [19/25]; IR: 65% [17/26]; HR: 68% [17/25]), improving overall survival (LR: 48% [12/25]; IR: 54% [14/26]; HR: 56% [14/25]), and delaying disease progression (LR: 40% [10/25]; IR: 50% [13/26]; HR: 48% [12/25]) were ranked 1 out of 3 as the most important goals for pts with MF. Most physicians (81% [21/26]) reported that ruxolitinib (RUX) increased the severity of anemia in pts with MF. PRFs were completed for 44 pts (South Korea: n=5, Taiwan: n=27, Canada: n=12). At data collection, the median (range) age was 67.0 (36.0-90.0) years and 45% (n=20) were female. At MF diagnosis, 34% of pts (15/44) had intermediate-2 risk MF, which was the category with the highest percentage of pts. At initial presentation of MF symptoms, 75% of pts (33/44) had anemia, with the median (range) Hb level being 8.0 (5-12) g/dL at anemia diagnosis. Of pts with MF, 26 received systemic drug treatment with the most common frontline treatment being RUX (77% [20/26]). Conclusions Most pts presented with anemia and most received RUX as their first line of treatment. Physicians agreed that reducing transfusion burden, improving Hb levels, and reducing fatigue are critical factors that determine the choice of therapy, especially as existing JAKi significantly increase these risks. Anemia complicates the management of MF and reiterates the need for tailored therapeutic approaches that addresses the underlying disease and the associated hematologic complications.

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