[Efficacy and safety analysis of a combination regimen with BCL-2 inhibitor in relapsed/refractory primary systemic light chain amyloidosis with t(11;14) from a single center].

Objective: To explore the efficacy and safety of BCL-2 inhibitor-based treatment in patients with relapsed/refractory t (11; 14) primary systemic light chain amyloidosis. Methods: This was a retrospective case series study. Ten patients with relapsed/refractory t(11;14) primary systemic light chain amyloidosis who had all received treatment with a combination regimen including the BCL-2 inhibitor venetoclax from January 2018 to November 2022 at the Hematology Department of Peking University People's Hospital were included. Adverse events, and hematological and organ responses were evaluated. Results: The median age of the ten enrolled patients was 59 (range 41-78) years, and the male to female ratio was 8∶2. Except for one patient, a very good partial or better response was achieved in 8/9 patients and one patient obtained a partial response. The overall response rate was 100%. The median time to achieve a hematological response was 60 (range 24-236) days. At least one organ response was observed in 7/9 patients. With a median follow-up of 18 months, one patient experienced hematological progression and one patient died. Grade 3 adverse events included lymphocytopenia (3 cases), anemia (1 case), diarrhea (1 case), and appendicitis (1 case). One patient died of pulmonary fungal infection two months after completion of treatment, which was not excluded as being treatment related. Conclusion: A combination regimen including BCL-2 inhibitors in patients with relapsed/refractory t(11;14) primary systemic light chain amyloidosis is a potentially safe and effective treatment option that warrants further investigation.目的： 分析BCL-2抑制剂为基础的治疗方案对伴t（11；14）的复发/难治性原发性系统性轻链型淀粉样变性患者的疗效及安全性。 方法： 回顾性病例系列研究。连续收集2018年1月至2022年11月在北京大学人民医院血液科应用含BCL-2抑制剂维奈克拉联合方案治疗的伴t（11；14）的复发/难治性原发性系统性轻链型淀粉样变性患者10例，分析其血液学及脏器疗效及不良反应发生情况。 结果： 10例患者的中位年龄59岁（范围41~78岁），男女比例为8∶2。除1例患者应用维奈克拉治疗不足1疗程，9例可评估疗效患者中8例血液学疗效≥非常好的部分缓解，1例获得部分缓解，总体缓解率100%。达到最佳血液学疗效的中位时间为60 d（范围24~236 d）。9例可评估疗效患者中7例至少1个脏器达到缓解率。总体中位随访18个月，1例患者血液学进展，1例患者死亡。≥3级不良反应为淋巴细胞减少3例、贫血1例、腹泻1例、阑尾炎1例，1例患者在结束治疗2个月后合并肺部真菌感染死亡，不除外与治疗相关。 结论： 含BCL-2抑制剂的联合方案治疗伴t（11；14）复发/难治性原发性系统性轻链型淀粉样变患者是一种有前景的安全有效的治疗选择，值得进一步研究。.