自体外周血造血干细胞移植治疗原发性轻链型淀粉样变性31例临床分析

Autologous peripheral blood hematopoietic stem cell transplantation for patients with primary light chain amyloidosis： experience of 31 cases in a single center

目的探讨自体外周血造血干细胞移植（ASCT）治疗原发性轻链型淀粉样变性的疗效和安全性。方法回顾性分析2009年1月至2015年6月接受ASCT治疗的31例原发性轻链型淀粉样变性患者的临床资料、疗效、生存及转归。结果31例患者中男18例、女13例，中位年龄55（43-66）岁；20例患者为单器官受累；80.6%的患者为Mayo 1期；诊断至ASCT的中位时间为3（0.5-26）个月；粒细胞植入和血小板植入的中位时间分别为11（9-12） d和11（8-14） d；无移植相关死亡病例。在可评价疗效的27例患者中，总体血液学缓解率为85.2%（23例），其中完全缓解率和非常好的部分缓解率分别为63.0%（17例）和7.4%（2例），获得最佳血液学缓解的中位时间为4（1-21）个月。总体器官缓解率为59.2%，获得器官缓解的中位时间为8（3-18）个月。中位随访21个月，1例患者死亡，3例患者出现疾病进展，预计的3年无进展生存率和总生存率分别为92.8%和96.4%。结论ASCT对于早期原发性轻链型淀粉样变性患者是一种有效、安全的治疗手段。

ObjectiveTo evaluate the efficacy and safety of autologous peripheral blood hematopoietic stem cell transplantation （ASCT） for patients with primary light chain （AL） amyloidosis.MethodsClinical data, hematological and organ response, safety and survival status of 31 patients with AL amyloidosis who had received ASCT from January 2009 to June 2015 were retrospectively analyzed.ResultsAmong 31 patients, there were 18 males and 13 females with the median age of 55 （range, 43-66） years old. Involvement of 1 organ was presented in 20 patients. 80.6% patients were defined as Mayo stage 1. The median time from diagnosis to ASCT was 3 （range, 0.5-26） months. The median time to neutrophil and platelet engraftment was 11 （range, 9-12） days and 11 （range, 8-14） days, respectively. No one patient had transplantation related death. Among 27 evaluable patients, overall best hematological response was 85.2% with complete response of 63.0% and very good partial response of 7.4%. The median time to the best hematological response was 4 （range, 1-21） months. 59.2% patients archived organ response and the median time to organ response was 8 （range, 3-18） months. After the median follow up time of 21 months, one patient had died and three patients had progressed. Therefore, the estimated 3 years progress free survival and overall survival was 92.8% and 96.4%, respectively. ConclusionsASCT was an effective and safe treatment for patients with primary AL amyloidosis in early stage.