Rüsing Lina Zoe, Schweighofer Jakob, Aschauer Julia, Jeryczynski Georg, Vospernik Lea, Gisslinger Heinz, Bumberger Armin Marcus, Cserna Julia, Riedl Julia, Agis Hermine, Krauth Maria-Theresa
Department of Medicine I, Medical University Vienna, 1090 Wien, Austria.
Department of Ophthalmology and Optometry, Medical University Vienna, 1090 Wien, Austria.
Cancers (Basel). 2025 Jul 19;17(14):2398. doi: 10.3390/cancers17142398.
: Belantamab mafodotin (belamaf) is a BCMA-targeting antibody-drug conjugate used in triple-class refractory multiple myeloma. Despite its efficacy, keratopathy remains a significant dose-limiting toxicity. Following its withdrawal from the U.S. market in 2022, its use in Austria is limited to clinical trials or compassionate use. : In this real-world, retrospective study, we analyzed 36 relapsed/refractory, BCMA-naïve multiple myeloma patients treated at the University Hospital of Vienna (January 2020-June 2024); 42% received a reduced dose (1.9 mg/kg) throughout all treatment cycles. The primary objective was to assess adverse events, particularly keratopathy, and the impact of dose modifications on toxicity and efficacy. : The overall response rate was 64%, with responders having significantly fewer prior therapy lines (median 3 vs. 4.5, = 0.015). Median PFS was 7.3 months, significantly longer in responders (11.1 vs. 1.6 months, < 0.0001); median OS was 20.1 months, also longer in responders (not reached vs. 18 months, = 0.031). Keratopathy occurred in 75% of patients; 33% experienced grade 3-4 events. Dose reduction significantly decreased grade 3-4 keratopathy (7% vs. 52%, = 0.004) and thrombocytopenia (33% vs. 67%, = 0.048) without compromising efficacy. : Belamaf dose reductions improved tolerability without loss of efficacy, supporting reduced dosing in practice.
贝兰他单抗莫福汀（belamaf）是一种用于治疗三重难治性多发性骨髓瘤的靶向B细胞成熟抗原（BCMA）的抗体药物偶联物。尽管其疗效显著，但角膜病变仍然是一种严重的剂量限制性毒性反应。自2022年退出美国市场后，其在奥地利的使用仅限于临床试验或同情用药。：在这项真实世界的回顾性研究中，我们分析了在维也纳大学医院接受治疗的36例复发/难治性、未接受过BCMA治疗的多发性骨髓瘤患者（2020年1月至2024年6月）；42%的患者在所有治疗周期中接受了减量（1.9mg/kg）。主要目的是评估不良事件，尤其是角膜病变，以及剂量调整对毒性和疗效的影响。：总体缓解率为64%，缓解者之前接受的治疗线数显著更少（中位数分别为3和4.5，P = 0.015）。中位无进展生存期（PFS）为7.3个月，缓解者显著更长（11.1个月对1.6个月，P < 0.0001）；中位总生存期（OS）为20.1个月，缓解者也更长（未达到对18个月，P = 0.031）。75%的患者发生了角膜病变；33%经历了3 - 4级事件。剂量减少显著降低了3 - 4级角膜病变（7%对52%，P = 0.004）和血小板减少症（33%对67%，P = 0.048），且不影响疗效。：贝兰他单抗莫福汀剂量减少提高了耐受性且不损失疗效，支持在实际应用中降低给药剂量。
