贝兰他单抗莫福汀治疗复发/难治性多发性骨髓瘤（RRMM）的真实世界疗效：西班牙扩大使用计划（EAP）的初步结果
Real-World Outcomes of Belantamab Mafodotin for Relapsed/Refractory Multiple Myeloma (RRMM): Preliminary Results of a Spanish Expanded Access Program (EAP).
作者信息
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
机构信息
Hospital Universitario La Princesa, Madrid, Spain.
Hospital Universitario 12 de Octubre, Madrid, Spain.
出版信息
Oncol Ther. 2023 Mar;11(1):83-96. doi: 10.1007/s40487-022-00212-5. Epub 2022 Dec 12.
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.
引言
贝兰他单抗马福多汀（BM）是一种新型抗B细胞成熟抗原（BCMA）抗体药物偶联物，最近被批准用于治疗三重难治性复发/难治性多发性骨髓瘤（RRMM）。我们评估了在西班牙扩大使用计划（EAP）下接受BM治疗的患者的真实世界疗效。
方法
我们进行了一项观察性、回顾性、多中心研究，纳入了接受≥1剂BM治疗的RRMM患者（2019年11月至2021年6月）。主要终点是总缓解率（ORR）。次要终点是无进展生存期（PFS）、总生存期（OS）和治疗中出现的不良事件（TEAE）发生率。
结果
纳入33例患者，中位年龄70岁（范围46 - 79岁）。从诊断到入组的中位时间为71个月（范围10 - 858个月）。既往治疗的中位线数为5（范围3 - 8线）；90%的患者对三种/四种/五种治疗方案耐药；48%表现为高危细胞遗传学特征。BM的中位剂量为3（范围1 - 16剂），中位随访时间为11个月（6 - 15个月）。ORR为42.2%（≥VGPR为18.2%）。总体人群的中位PFS为3个月（95%CI 0.92 - 5.08），达到≥PR的患者为11个月（HR 0.26；95%CI 0.10 - 0.68）。PFS在年龄、细胞遗传学风险和既往治疗线数方面无显著差异。OS为424天（95%CI 107 - 740）。非血液学TEAE（57.6%的患者；30.3%≥3级）包括角膜病变（51.5%；21.2%≥3级）和患者报告的视力相关症状（45.5%）。70.6%的患者角膜病变得到缓解。3级血液学TEAE为18.2%，血小板减少症为21.2%。因TEAE导致的剂量减少：30.3%；治疗延迟：36.4%。治疗中断原因：疾病进展（54.5%）、毒性（非眼部；6%/眼部；6%/眼部 + 非眼部毒性；3%）、死亡（6%）和患者决定（3%）。
结论
BM在RRMM中显示出显著的抗骨髓瘤活性，安全性可控。这些结果证实了在BM关键试验中观察到的结果。
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