Real-World Outcomes of Belantamab Mafodotin for Relapsed/Refractory Multiple Myeloma (RRMM): Preliminary Results of a Spanish Expanded Access Program (EAP)

• Published in: Oncology and therapy 2023-03, Vol.11 (1), p.83-96
• Main Authors: Alegre, Adrián, Benzo, Gonzalo, Alonso, Rafael, Martínez-López, Joaquín, Jimenez-Ubieto, Ana, Cuéllar, Clara, Askari, Elham, Prieto, Elena, Aláez, Concepción, Aguado, Beatriz, Velasco, Alberto, Krsnik, Isabel, Bocanegra, Ana, Llorente, Laura, Muñoz-Linares, Cristina, Morales, Ana, Giménez, Eugenio, Iglesias, Rebeca, Martínez-Chamorro, Carmen, Alonso, Aránzazu, Jiménez-Montes, Carmen, Blanchard, María J.
• Format: Article
• Language: English
• Subjects: Belantamab mafodotin; Effectiveness; Internal Medicine; Medicine; Medicine & Public Health; Original Research; Real-world outcomes; Safety; Triple-class relapsed and refractory multiple myeloma
• ISSN: 2366-1070; 2366-1089; 2366-1089
• DOI: 10.1007/s40487-022-00212-5

Introduction Belantamab mafodotin (BM) is a new anti-BCMA antibody–drug conjugate, recently approved for triple-class relapsed and refractory multiple myeloma (RRMM). We assessed real-world outcomes with BM in patients under the Spanish Expanded Access Program (EAP). Methods We conducted an observational, retrospective, multicenter study including RRMM patients who received ≥ 1 dose of BM (Nov 2019 to Jun 2021). The primary endpoint was overall response rate (ORR). Secondary endpoints were progression-free survival (PFS), overall survival (OS), and incidence of treatment-emergent adverse events (TEAEs). Results Thirty-three patients were included with a median of 70 years of age (range, 46–79 years). Median time from diagnosis was 71 months (range, 10–858 months). Median prior lines was 5 (range, 3–8 lines); 90% of patients were triple-/quad-/penta-refractory; 48% showed high-risk cytogenetics. Median BM doses was 3 (range 1–16 doses), with a median follow-up of 11 months (6–15 months). ORR was 42.2% (≥ VGPR, 18.2%). Median PFS was 3 months (95% CI 0.92–5.08) in the overall population, and 11 months (HR 0.26; 95% CI 0.10–0.68) for patients who achieved ≥ PR. PFS was not significantly different according to age, cytogenetic risk, and prior therapy lines. OS was 424 days (95% CI 107–740). Non-hematological TEAEs (57.6% of patients; 30.3% ≥ G3) included keratopathy (51.5%; 21.2% ≥ G3) and patient-reported vision-related symptoms (45.5%). Keratopathy was resolved in 70.6% of patients. G3 hematological TEAEs was 18.2%, thrombocytopenia (21.2%). Dose reductions due to TEAEs: 30.3%; delays: 36.4%. Treatment discontinuation causes: progression (54.5%), toxicity (non-ocular; 6%/ocular; 6% /ocular + non-ocular toxicity; 3%), death (6%), and patient’s decision (3%). Conclusions BM showed relevant anti-myeloma activity in RRMM with a manageable safety profile. These results corroborate those observed in the BM pivotal trial.