Objective: To evaluate the efficacy and safety of autologous peripheral blood hematopoietic stem cell transplantation (ASCT) for patients with primary light chain (AL) amyloidosis.
Methods: Clinical 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.
Results: Among 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.
Conclusions: ASCT was an effective and safe treatment for patients with primary AL amyloidosis in early stage.
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。