Abstracts Clinical Lymphoma, Myeloma & Leukemia September 2023 S370 physicians provided data on 22 patients with PNH receiving pegcetacoplan for a median (SD) 10.3 (3.0) months. 90.9% had switched from a C5 inhibitor. Median (SD) age was 35.0 (10.8), and 59.1% were male. Median (SD) time since diagnosis was 2.4 (2.4) years. From pegcetacoplan initiation to point of study, physicians reported a 3.4 g/dL improvement in hemoglobin (8.2 g/dL vs 11.6 g/dL, respectively), improvement in lactate dehydrogenase (LDH) (27.3% vs 68.2% reporting LDH <1.5x ULN when ULN was set to 250 U/L, respectively) and an increased proportion of patients experiencing no fatigue (0.0% vs 59.1%, respectively). All patients were considered by their physicians to have ‘well/very well controlled’ disease at time of survey and were ‘satisfied/completely satisfied’ with their patients’ treatment. Physicians reported 86.4% of patients had ‘excellent/very good’ overall quality of life. Conclusions: These findings demonstrate the real-world effectiveness of pegcetacoplan through improvement in endpoints, in line with clinical trial findings. Satisfaction with treatment and disease control were reported favorably, highlighting the potential for pegcetacoplan to improve outcomes of current patients with PNH. Further research is required to understand the long-term effectiveness of pegcetacoplan in the real-world. Keywords: PNH, pegcetacoplan, C3 inhibitor, real-world, survey MDS-567 Real‑World Analysis of a Large Electronic Medical Record Database of Patients With Higher‑Risk Myelodysplastic Neoplasms (HR MDS): Treatment Profiles, Clinical Effectiveness, and Key Adverse Event Rates Rohan Shah BS1, Anusorn Thanataveerat DrPH, MPH, BS1, Ricky Rudraraju PhD2, Kelly Curtis MD2, Teraneh Z. Jhaveri PhD2, Gulce Askin MPH, BA1, Debra E. Irwin PhD, MSPH, BS1, Jeanne Pierzynski PhD, MPH, BS2, Cosmina Hogea PhD, MS, BS2, Eduardo J. Sabate MD, MPH2, Arman P. Sali MS, BA2, Mellissa Williamson PhD, MPH, BSc2 1Aetion, Inc, Real World Evidence Solutions, New York, USA. 2Gilead Sciences, Inc, Foster City, USA Context: Patients diagnosed with HR MDS experience suboptimal outcomes with currently available treatments. Characterizing care and unmet needs of HR MDS patients in a real-world context is needed to assess the potential impact of novel therapies. Objective: To describe demographics and clinical characteristics, treatment profiles, key adverse events, and clinical outcomes of newly diagnosed HR MDS patients. Design: This descriptive, retrospective cohort study used ConcertAI’s electronic medical records database from a large network of oncology practices in the US. Patients: From January 1, 2010 to October 21, 2022, a total of 2028 HR MDS patients (≥18 years old) were identified and indexed into treatment cohorts (monotherapy hypomethylating agents [HMAs], immunosuppressive therapy [IST], venetoclax±HMAs [VEN], lenalidomide [LEN], hematopoietic stem cell transplant [HSCT], and supportive care only [SC]). Outcome Measures: Overall survival (OS), time-to-acute myeloid leukemia (AML) transformation, event rates per 100 person-years (95% confidence interval [CI]) of incident anemia, thromboembolic events, cardiac events, and infections were assessed. Results: The median age was 70 years at diagnosis (~60% male). Of the first-line treatments, HMAs were the most common (59%), while 10% received VEN off-label. Most patients in each treatment cohort (55%–76%), except HSCT and IST, received care in the community setting. Patients who received HSCT were the youngest (median=60 years) while those who received SC were the oldest (median=80 years). Event incident rates were: anemia 1235.2 (1147.6–1322.8), cardiac arrhythmia 21.5 (18.9–24.2), cardiac failure 10.7 (8.9–12.5), and infection 42.1 (38.1–46.1). Overall, 75% of patients died during follow-up (median OS=296 days), with median OS in the HSCT cohort (652 days) over twice that of the overall cohort. Over 40% of patients transformed to AML (median time-to-transformation=346 days [95% CI, 302-403]), with the highest percentage (47%) and shortest time (median=44 days) occurring in the VEN cohort. Keywords: real-world data, myelodysplastic neoplasm, MDS, clinical outcome, outcome MDS-569 Real‑World Use of Hypomethylating Agents (HMA)/Venetoclax Combinations in Patients With Myelodysplastic Neoplasms (MDS) in the Arabian Gulf Region Fatima Khadadah MD1, Honar Cherif MD2, Halima AlOmri MD3, Ahmad Alhuraiji MD1, Ramesh Pandita MD1 1Dept of Hematology, Kuwait Cancer Cente, Kuwait, Kuwait. 2National Center for Cancer Care & Research Hamad Medical Corporation, Doha, Qatar. 3National Center for Cancer Care & Research Hamad Medical Corporation, Qatar Context: Retrospective data thus far has supported improved responses with HMA/venetoclax combinations in both the frontline and relapsed settings. A Phase Ib trial of azacitidine and venetoclax for high-risk MDS patients demonstrated an overall response rate (ORR) of 80%. Retrospective data from the Moffitt Centre comparing HMA/venetoclax to patients who received HMA showed an ORR of 77% compared to 40% for patients who only received HMA. Objectives: Our aim was to obtain real-world evidence for use of combination HMA/venetoclax to treat myelodysplastic patients in the Arabian Gulf region. Methods: This is a retrospective study of patients with myelodysplastic neoplasm treated with a HMA/ venetoclax in 2 different countries from Jan 2020–May 2023. Cases were included if they were over the age of 18 and had a diagnosis of MDS or MDS/AML. Both frontline and relapsed/refractory patients were included. Patients were excluded if they had more than 20% blasts or received an additional agent with their treatment. Results: Data on 13 patients was collected in Kuwait and Qatar. All patients had high-risk MDS, MDS/AML. Median follow up was 14 months (range 4–23). Azacitidine was the only HMA used. Venetoclax was given for a median of 14.5 days (range 10–28). All patients received concurrent azole prophylaxis. A median of 6 cycles were received
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