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
10.3760/cma.j.cn112138-20230224-00109
- VernacularTitle:含BCL-2抑制剂的联合方案治疗伴t（11；14）的复发/难治性原发性系统性轻链型淀粉样变性的疗效及安全性分析
- Author:
Aili ZHAI
1
;
Yang LIU
;
Nan PENG
;
Lizhong GONG
;
Xuelin DOU
;
Lei WEN
;
Jin LU
Author Information
1. 北京大学人民医院 北京大学血液病研究所 国家血液系统疾病临床医学研究中心，北京 100044
- Keywords:
Amyloidosis;
Drug therapy;
t (11;
14);
Venetoclax
- From:
Chinese Journal of Internal Medicine
2023;62(11):1323-1328
- CountryChina
- Language:Chinese
-
Abstract:
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.
