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

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. CONCLUSION: ASCT was an effective and safe treatment for patients with primary AL amyloidosis in early stage.