Primary systemic amyloidosis: comparison of melphalan and prednisone versus placebo.
Primary systemic amyloidosis: comparison of melphalan and prednisone versus placebo.
复制标题
原发性系统性淀粉样变性：马法兰和泼尼松与安慰剂的比较。
作者：
R. Kyle;P. Greipp
原发性淀粉样变性尚不存在令人满意的治疗方法。由于淀粉样原纤维由单克隆轻链的一部分组成，因此用可有效对抗合成单克隆轻链的浆细胞的烷化剂治疗淀粉样变性似乎是合理的。 55 名原发性系统性淀粉样变性患者被随机（双盲）接受马法兰-泼尼松或安慰剂治疗。与安慰剂组相比，接受马法兰-泼尼松治疗的患者能够继续治疗更长的时间，并在密码被破解之前接受更大的剂量。在这组患者中，2 名患者的肾病综合征消失，另外 8 名患者的尿蛋白排泄量减少了 50% 以上。 13 名接受马法兰-泼尼松治疗超过 12 个月的患者中，6 名病情好转，3 名病情稳定，4 名病情进展。各组之间的生存率没有显着差异。
Satisfactory treatment for primary amyloidosis does not exist. Because the amyloid fibrils consist of a portion of a monoclonal light chain, it appears reasonable to treat amyloidosis with alkylating agents that are effective against the plasma cells that synthesize monoclonal light chains. Fifty-five patients with primary systemic amyloidosis were randomized (double blind) to melphalan-prednisone or placebo. In comparison with the placebo group, patients given melphalan-prednisone were able to continue on treatment for a longer time and to receive larger doses before the code was broken. Among this group, the nephrotic syndrome disappeared in two patients and urinary excretion of protein was reduced by more than 50% in eight others. Of 13 patients who received melphalan-prednisone for more than 12 mo, 6 improved, 3 were stable, and 4 had progression of disease. Survival did not differ significantly between the groups.