以达雷妥尤单抗为基础的化疗方案治疗系统性轻链型淀粉样变性真实世界研究  

A real-world study of daratumumab-based chemotherapy regimens for systemic light chain amyloidosis

作　　者：王珺 吴佳霏 王依景 郑博月 王宇 江川艳 刘驰 李慧[1] Wang Jun;Wu Jiafei;Wang Yijing;Zheng Boyue;Wang Yu;Jiang Chuanyan;Liu Chi;Li Hui(Department of Hematology,Sichuan Academy of Medical Sciences,Sichuan Provincial People's Hospital,Chengdu 610072,China;School of Clinical Medicine,University of Electronic Science and Technology of China,Chengdu 610056,China;Department of Hematology,Chengdu Second People's Hospital,Chengdu 610021,China;Department of Nephrology,Sichuan Academy of Medical Sciences,Sichuan Provincial People's Hospital,Chengdu 610072,China)

机构地区：[1]四川省医学科学院四川省人民医院血液科,成都610072 [2]电子科技大学医学院临床医学院,成都610056 [3]成都市第二人民医院血液科,成都610021 [4]四川省医学科学院四川省人民医院肾内科,成都610072

出　　处：《白血病．淋巴瘤》2023年第10期594-599,共6页Journal of Leukemia & Lymphoma

基　　金：四川省自然科学基金(23NSFSC0607);四川省医学科学院四川省人民医院基金(2021LY16);四川省干保课题(川干研2021-213);白求恩·血液科研能力建设项目(J202201E023)。

摘　　要：目的探讨达雷妥尤单抗治疗系统性轻链(AL)型淀粉样变性的疗效和安全性。方法回顾性分析2020年1月至2022年11月于四川省人民医院接受以达雷妥尤单抗为基础的化疗方案治疗的24例AL型淀粉样变性患者的临床资料。总结患者治疗情况,评估疗效,采用Kaplan-Meier法进行生存分析,分析不良反应发生情况。结果24例患者中,梅奥2004分期Ⅰ期2例(8.33%),Ⅱ期2例(8.33%),Ⅲ期20例(83.33%)。所有患者均采用以达雷妥尤单抗为基础的方案进行治疗,可评估疗效17例。15例患者应用DVd(达雷妥尤单抗+硼替佐米+地塞米松)方案治疗,7例应用DVCd(达雷妥尤单抗+硼替佐米+环磷酰胺+地塞米松)方案治疗,1例应用DRd(达雷妥尤单抗+来那度胺+地塞米松)方案治疗,1例应用DTd(达雷妥尤单抗+沙利度胺+地塞米松)方案治疗。17例可评估疗效患者应用以达雷妥尤单抗为基础的方案治疗1个疗程后,严格意义的完全缓解(sCR)率为41.18%(7/17),总反应率(ORR)为88.24%(15/17)。17例一线应用以达雷妥尤单抗为基础的化疗方案治疗的患者中,11例可评估疗效者治疗1个疗程后sCR率为36.36%(4/11),ORR为90.90%(10/11)。5例复发难治患者中,4例可评估疗效者治疗1个疗程后sCR率为50.00%(2/4),ORR为75.00%(3/4)。24例患者中,初诊时17例肾脏受累,应用以达雷妥尤单抗为主的方案治疗1个疗程后,7例可评估疗效患者的ORR达85.71%(6/7),其中42.86%(3/7)患者肾脏缓解达非常好的部分缓解(VGPR)及以上;初诊时19例心脏受累,可评估疗效的14例患者ORR达85.71%(12/14),其中42.86%(6/14)患者心脏缓解达VGPR及以上。应用以达雷妥尤单抗为基础的化疗方案治疗后,患者的主要不良反应为输液相关不良反应、骨髓抑制及感染,均可耐受。24例患者中位随访7.0个月(0.5~16.5个月),中位无进展生存(PFS)时间为7.0个月(0.5~16.5个月),中位总生存(OS)时间为7.0个月(0.5~35.0个月)。结论以达雷妥尤单�Objective To investigate the efficacy and safety of daratumumab in the treatment of systemic light chain amyloidosis.Methods The clinical data of 24 patients with systemic light chain amyloidosis who received daratumumab-based regimens in Sichuan Provincial People's Hospital from January 2020 to November 2022 were retrospectively analyzed.The treatment process of patients was summarized and the therapeutic efficacy was evaluated.Kaplan-Meier method was used to make survival analysis and the adverse reactions were analyzed.Results All 24 patients included 2 cases(8.33%)of Mayo 2004 stageⅠ,2 cases(8.33%)of Mayo 2004 stageⅡand 20 cases(83.33%)of Mayo 2004 stageⅢ.All patients were treated with daratumumab-based regimen,and 17 patients had evaluable efficacy.In the chemotherapy regimens,15 patients received DVd(daratumumab+bortezomib+dexamethasone)regimen,7 patients received DVCd(daratumumab+bortezomib+cyclophosphamide+dexamethasone)regimen,1 patient received DRd(daratumumab+lenalidomide+dexamethasone)regimen,and 1 patient received DTd(daratumumab+thalidomide+dexamethasone)regimen.After 1 course of daratumumab-based regimens in 17 cases with evaluable efficacy,the strict complete remission(sCR)rate was 41.18%(7/17),the overall response rate(ORR)was 88.24%(15/17).Among 17 patients who received daratumumab-based chemotherapy regimen as the first-line treatment,sCR rate of 11 cases with evaluable efficacy was 36.36%(4/11)after 1 course of treatment ORR was 90.90%(10/11).Among 5 relapsed/refractory patients,sCR rate of 4 cases with evaluable efficacy was 50.00%(2/4)after 1 course of treatment;ORR was 75.00%(3/4).Among 24 patients,renal involvement was found in 17 patients at the initial diagnosis.After 1 course of daratumumab-based chemotherapy regimen,ORR of 7 cases with evaluable efficacy was 85.71%(6/7),among which 42.86%(3/7)patients with renal involvement had an assessed renal response of very good partial remission(VGPR)or above.At the initial diagnosis,19 cases had cardiac involvement;ORR of 14 cases with eva

关 键 词：淀粉样变性 免疫球蛋白轻链 达雷妥尤单抗 治疗结果 

分 类 号：R597.2[医药卫生—内科学]