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贝兰他单抗莫福汀治疗复发/难治性轻链型淀粉样变性:真实世界多中心经验及文献综述

Belantamab Mafodotin in Relapsed/Refractory AL Amyloidosis: Real-World Multi-Center Experience and Review of the Literature.

作者信息

Lebel Eyal, Vainstein Vladimir, Milani Paolo, Palladini Giovanni, Shragai Tamir, Lavi Noa, Magen Hila, Assayag Miri, Avivi Irit, Gatt Moshe E

机构信息

Faculty of Medicine, The Hebrew University of Jerusalem, Jerusalem, Israel.

Hematology Department, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.

出版信息

Acta Haematol. 2025;148(4):419-426. doi: 10.1159/000541594. Epub 2024 Oct 2.

Abstract

INTRODUCTION

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.

METHODS

We report a multi-center cohort of AL patients receiving BLM, and review all previous data on BLM therapy in AL.

RESULTS

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.

CONCLUSION

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.

摘要

引言

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。

方法

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。

结果

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。

结论

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。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3d6c/12306947/f8b57909826d/aha-2025-0148-0004-541594_F01.jpg

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