美法仑联合大剂量地塞米松一线治疗原发性轻链型淀粉样变的疗效分析  

Oral melphalan plus high-dose dexamethasone as first-line therapy for patients with primary light chain amyloidosis

作　　者：张聪丽 沈恺妮 冯俊 曹欣欣 毛玥莹 张路 苏薇[2] 周道斌 李剑 Zhang Congli;Shen Kaini;Feng Jun;Cao Xinxin;Mao Yueying;Zhang Lu;Su Wei;Zhou Daobin;Li Jian(Department of Hematology,Peking Union Medical College Hospital,Chinese Acad-emy of Medical Sciences ＆ Peking Union Medical College,Beijing 100730,China)

机构地区：[1]中国医学科学院、北京协和医学院北京协和医院血液内科,100730 [2]中国医学科学院、北京协和医学院北京协和医院检验科,100730

出　　处：《中华血液学杂志》2018年第8期641-644,共4页Chinese Journal of Hematology

摘　　要：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。ObjectiveTo evaluate the response of oral melphalan plus high-dose dexamethasone （MDex） for patients with primary light chain amyloidosis （pAL）. MethodsClinical 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. ResultsOf 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. ConclusionsMDex was an effective treatment for patients with early stage pAL, but was not suitable for those with severe cardiac involvement.

关 键 词：美法仑 地塞米松 淀粉样变 轻链型 治疗结果 

分 类 号：R597.2[医药卫生—内科学]