Lebel Eyal, Kastritis Efstathios, Palladini Giovanni, Milani Paolo, Theodorakakou Foteini, Aumann Shlomzion, Lavi Noa, Shargian Liat, Magen Hila, Cohen Yael, Gatt Moshe E, Vaxman Iuliana
Faculty of Medicine, The Hebrew University of Jerusalem, Jerusalem 91120, Israel.
Hematology Department, Hadassah-Hebrew University Medical Center, Jerusalem 91120, Israel.
Cancers (Basel). 2023 Mar 10;15(6):1710. doi: 10.3390/cancers15061710.
Therapeutic options in relapsed refractory (R/R) light-chain (AL) amyloidosis patients are limited. Given the encouraging results in t(11;14) multiple myeloma and the high prevalence of t(11;14) in AL amyloidosis, venetoclax is an attractive treatment option in this setting. We report here the results of a multi-center retrospective study on 26 R/R AL amyloidosis patients treated off-label with venetoclax. The median lines of therapy prior to venetoclax was 3.5 (range 1-7), and 88% of our cohort had t (11;14). Twenty-two patients (85%) were previously treated with daratumumab. The overall hematologic response rate was 88%, 35% achieved a CR, and 35% achieved VGPR. The median event-free survival was 25 months (m) (95% CI 9.7 m-not reached), and the median overall survival was 33 m (95% CI 25.9-39.2 m). Most of the patients in this cohort are in ongoing deep responses and continuing venetoclax therapy. The treatment was relatively safe. One patient died due to infection, and there were two grade 3 infections in our cohort. Tumor lysis syndrome (TLS) was not seen in any patient. Dose reductions were frequent but did not affect the efficacy. These promising results require confirmation in a randomized controlled trial.
复发难治性（R/R）轻链（AL）淀粉样变性患者的治疗选择有限。鉴于在t(11;14)多发性骨髓瘤中取得的令人鼓舞的结果以及AL淀粉样变性中t(11;14)的高发生率，维奈克拉在这种情况下是一种有吸引力的治疗选择。我们在此报告一项针对26例接受维奈克拉标签外治疗的R/R AL淀粉样变性患者的多中心回顾性研究结果。维奈克拉治疗前的中位治疗线数为3.5（范围1 - 7），我们队列中的88%患者有t(11;14)。22例患者（85%）曾接受达雷妥尤单抗治疗。总体血液学缓解率为88%，35%达到完全缓解（CR），35%达到非常好的部分缓解（VGPR）。中位无事件生存期为25个月（m）（95%置信区间9.7 m - 未达到），中位总生存期为33 m（95%置信区间25.9 - 39.2 m）。该队列中的大多数患者处于持续深度缓解并继续接受维奈克拉治疗。治疗相对安全。1例患者死于感染，我们队列中有2例3级感染。未在任何患者中观察到肿瘤溶解综合征（TLS）。剂量减少频繁，但不影响疗效。这些有前景的结果需要在随机对照试验中得到证实。
