MDS-199 Real World Efficacy of Luspatercept in Patients with Lower-risk Myelodysplastic Syndromes/Neoplasms (LR-MDS); a Single Center Study in a Heavily Pretreated Cohort

• Published in: Clinical lymphoma, myeloma and leukemia myeloma and leukemia, 2024-09, Vol.24, p.S389-S389
• Main Authors: Kometas, Marisa, Hyak, Jonathan, Hoff, Fieke, Irizarry-Gatell, Vivian, Marinos, Alejandro, Jackson, Clayton, Anderson, Julia, Kalkan, Fatma, Ramakrishnan, Praveen, Afrough, Aimaz, Wolfe, Heather, Khan, Adeel, Anderson, Larry, Awan, Farrukh, Kaur, Gurbakhash, Yilmaz, Elif, Alvarenga, Julio, Chen, Weina, Weinberg, Olga, Cantu, Miguel, Collins, Robert H., Chung, Stephen S., Madanat, Yazan F.
• Format: Article
• Language: English
• Subjects: lower-risk myelodysplastic syndrome/neoplasms; luspatercept; MDS
• ISSN: 2152-2650
• DOI: 10.1016/S2152-2650(24)01349-1

Anemia, the most common cytopenia in patients with LR-MDS, leads to transfusion dependence (TD) and inferior outcomes. Luspatercept received regulatory approval for treatment of TD LR-MDS. Our study evaluated the real-world efficacy and safety of luspatercept in LR-MDS and the impact of baseline characteristics and prior treatments on hematologic response (HI-E). This retrospective, single-institutional study was performed at an academic tertiary referral center and included patients with LR-MDS treated with luspatercept from 01/2020 through 12/2023. Baseline characteristics extracted were prior therapies, serum erythropoietin (EPO) level, ring sideroblast (RS) status, karyotype, and gene mutations. Baseline transfusion burden (no=NTD, low=LTB, high=HTB) for all patients, HI-E in patients with ≥16 weeks of therapy, and progression-free survival were determined per IWG 2018 criteria. The cohort included 37 patients; median age was 74 years, 51% were female, 78% had RS, 73% were SF3B1-mutated, 32% had high-risk mutations, and 44% had serum EPO ≥200 mU/mL. Sixteen percent had therapy-related MDS, with 16% receiving 2 and 5% receiving 3 prior therapies. Per IPSS-M, 19% had higher-risk disease. Per IWG 2018, 19% were NTD, 41% were LTB, and 41% were HTB. Of 31 patients who were response evaluable, 52% had HI-E, and 48% of TD patients achieved ≥16 weeks of transfusion independence (TI). Median TI duration (48 weeks) did not differ between HTB and LTB responders. EPO