Skinner M, Anderson J, Simms R, Falk R, Wang M, Libbey C, Jones L A, Cohen A S
Arthritis Center, Thorndike Memorial Laboratories, Boston City Hospital, Massachusetts, USA.
Am J Med. 1996 Mar;100(3):290-8. doi: 10.1016/s0002-9343(97)89487-9.
A clinical trial designed to test whether treatment with melphalan, prednisone, and colchicine (MPC) is superior to colchicine (C) alone was performed in patients with primary amyloidosis (AL), a nonmalignant plasma cell dyscrasia.
Patients were randomized to MPC or C with stratification according to sex, time from diagnosis to study entry (ie, less than 3 months or 3 to 12 months), and dominant organ system involvement (ie, cardiac, renal, neurologic, or others). Data were gathered monthly from patients, quarterly from physicians, and annually in the Clinical Research Center. One hundred consecutive patients with AL amyloidosis admitted between 1987 and 1992 who met eligibility requirements were treated and followed for a minimum of 18 months. Fifty patients (group A) received daily oral colchicine and 50 patients (group B) received cycles of oral melphalan and prednisone every 6 weeks for 1 year as well as colchicine.
The principal outcome measure was median survival, which was compared in the two treatment groups and in the subgroups. The overall survival of all patients from study entry was 8.4 months. Comparing group A (C) to group B (MPC), the survival was 6.7 months versus 12.2 months (P = 0.087). Both treatment groups had poor survival for patients in the cardiac subgroup, longest survival in the renal group, and significant differences favoring MPC treatment only in patients whose major system manifestations were neurologic (P = 0.037) or other (P = 0.007). Multivariate analysis showed a strongly significant treatment effect (P = 0.003) and improved survival associated with not having cardiac or gastrointestinal involvement.
MPC was advantageous for patients whose major manifestations of amyloid disease were other than cardiac or renal. Better survival regardless of treatment was noted in patients for whom a satisfactory supportive treatment such as transplant or dialysis exists for their organ failure.
在原发性淀粉样变性（AL）患者中开展了一项临床试验，以测试美法仑、泼尼松和秋水仙碱联合治疗（MPC）是否优于单独使用秋水仙碱（C），原发性淀粉样变性是一种非恶性浆细胞增殖性疾病。
根据性别、从诊断到进入研究的时间（即少于3个月或3至12个月）以及主要器官系统受累情况（即心脏、肾脏、神经或其他）进行分层，将患者随机分为MPC组或C组。每月从患者处收集数据，每季度从医生处收集数据，并每年在临床研究中心收集数据。1987年至1992年间收治的100例符合入选标准的AL淀粉样变性患者接受治疗并随访至少18个月。50例患者（A组）每日口服秋水仙碱，50例患者（B组）每6周接受1年的口服美法仑和泼尼松治疗以及秋水仙碱治疗。
主要结局指标为中位生存期，对两个治疗组及亚组进行了比较。所有患者从进入研究开始的总生存期为8.4个月。将A组（C组）与B组（MPC组）进行比较，生存期分别为6.7个月和12.2个月（P = 0.087）。两个治疗组中心脏亚组患者的生存期均较差，肾脏组患者的生存期最长，仅在主要系统表现为神经（P = 0.037）或其他（P = 0.007）的患者中，MPC治疗具有显著差异。多因素分析显示治疗效果具有高度显著性（P = 0.003），且生存期改善与无心脏或胃肠道受累相关。
对于淀粉样疾病主要表现不是心脏或肾脏的患者，MPC治疗具有优势。对于其器官衰竭存在移植或透析等满意支持治疗的患者，无论接受何种治疗，生存期均较好。
