Objective: To evaluate the response of oral melphalan plus high-dose dexamethasone (MDex) for patients with primary light chain amyloidosis (pAL). Methods: Clinical data, hematological and organ responses, and survival of 76 patients with pAL who had received MDex from January 2009 to July 2017 were retrospectively analyzed. Results: Of 76 patients (47 males and 29 females with the median age of 56 [range, 20-74] years old), 19.70% patients were defined as Mayo 2004 stage 3, involvement of more than or two organs was presented in 65 (85.53%) patients. Among 60 response evaluable patients, overall hematological response was 48.33% with complete response of 20.00% and very good partial response of 20.00%, respectively. The median time to the hematological response was 5 (range, 1-15) months. 36.67% patients achieved organ response. After the median follow up of 23(range, 1-113) months for surviving patients, median progression-free survival (PFS) and overall survival (OS) were 34 and 43 months, respectively. In a three months landmark analysis, the median rates of PFS and OS were 46 and 65 months, respectively. The median OS rates of patients with Mayo 2004 stage 3 and non Mayo 2004 stage 3 were 5 and 65 months (P=0.001), respectively. Conclusions: MDex was an effective treatment for patients with early stage pAL, but was not suitable for those with severe cardiac involvement.
目的： 探讨口服美法仑联合大剂量地塞米松（MDex）方案一线治疗原发性轻链型淀粉样变（pAL）的疗效。 方法： 收集2009年1月至2017年7月接受MDex方案治疗的76例初治pAL患者临床资料，对其疗效及生存进行回顾性分析。 结果： ①76例患者中，男47例，女29例，中位年龄为56（20~74）岁。66例患者可进行梅奥2004分期（1、2、3期者分别为21、32、13例），65例（85.53%）患者存在≥2个器官受累。中位疗程数为5（1~21）个。②在可评价疗效的60例患者中，总体血液学缓解率为48.33%（29/60），完全缓解率和非常好的部分缓解率均为20.00%（12/60），获得最佳血液学缓解的中位时间为5（1~15）个月；总体器官缓解率为36.67%（22/60）。③生存患者的中位随访时间为23（1~113）个月，中位无进展生存（PFS）及总生存（OS）时间及分别为34、43个月。治疗3个月后仍存活患者的中位PFS和OS时间分别为46、65个月。④梅奥2004分期3期和1~2期患者的中位OS时间分别为5、65个月，差异有统计学意义（P=0.001）。 结论： MDex方案是pAL早期患者一种有效的治疗手段，但不适用于心脏受累的晚期患者。.
Keywords: Amyloidosis, light chain; Dexamethasone; Melphalan; Treatment outcome.