Lebel Eyal, Vainstein Vladimir, Milani Paolo, Palladini Giovanni, Shragai Tamir, Lavi Noa, Magen Hila, Assayag Miri, Avivi Irit, Gatt Moshe E
Faculty of Medicine, The Hebrew University of Jerusalem, Jerusalem, Israel.
Hematology Department, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.
Acta Haematol. 2025;148(4):419-426. doi: 10.1159/000541594. Epub 2024 Oct 2.
Treatment for relapsed/refractory AL amyloidosis (AL) is an unmet need. The safety and efficacy of belantamab mafodotin (BLM) in multiple myeloma are known, whereas in AL data are limited.
We report a multi-center cohort of AL patients receiving BLM, and review all previous data on BLM therapy in AL.
Twelve patients with a median of 3 (range 2-9) prior lines of therapy were included. The overall hematological response rate (ORR) was 75% (9/12), including 5 complete responses. Six of the 10 evaluable patients had organ responses. The median event-free survivals/overall survivals were 22.3 and 28.8 months, respectively. Grade 3 toxicities were mostly infections and keratopathy, occurring in 7/12 (58%). Hematological toxicities were rare. No grade 4/5 toxicities occurred. The review of the previous series reveals BLM provides an ORR of 60-83% with similar rates of corneal toxicity.
BLM, being an off-the-shelf therapy, with acceptable toxicity even in frail patients, may be a valuable option in AL, with a high ORR, and a signal for durable responses and high-quality organ responses.
复发/难治性轻链型淀粉样变（AL）的治疗需求尚未得到满足。贝兰他单抗莫福汀（BLM）在多发性骨髓瘤中的安全性和有效性已为人所知，而在AL中的数据有限。
我们报告了一组接受BLM治疗的AL患者的多中心队列研究，并回顾了此前关于BLM治疗AL的所有数据。
纳入了12例患者，这些患者之前接受治疗的中位数为3线（范围2 - 9线）。总体血液学缓解率（ORR）为75%（9/12），包括5例完全缓解。10例可评估患者中有6例出现器官缓解。无进展生存期/总生存期的中位数分别为22.3个月和28.8个月。3级毒性反应主要为感染和角膜病变，7/12（58%）的患者出现此类情况。血液学毒性反应罕见。未发生4/5级毒性反应。对既往系列研究的回顾显示，BLM的ORR为60 - 83%，角膜毒性发生率相似。
BLM作为一种现成可用的治疗方法，即使对体弱患者也具有可接受的毒性，可能是AL治疗中的一个有价值的选择，具有较高的ORR，且有持久缓解和高质量器官缓解的迹象。
