Zemer D, Pras M, Sohar E, Modan M, Cabili S, Gafni J
N Engl J Med. 1986 Apr 17;314(16):1001-5. doi: 10.1056/NEJM198604173141601.
To determine whether colchicine prevents or ameliorates amyloidosis in patients with familial Mediterranean fever, we followed 1070 patients with the latter disease for 4 to 11 years after they were advised to take colchicine to prevent febrile attacks. Overall, at the end of the study, the prevalence of nephropathy was one third of that in a study conducted before colchicine was used to treat familial Mediterranean fever. Among 960 patients who initially had no evidence of amyloidosis, proteinuria appeared in 4 who adhered to the prophylactic schedule and in 16 of 54 who admitted non-compliance. Life-table analysis showed that the cumulative rate of proteinuria was 1.7 percent (90 percent confidence limits, 0.0 and 11.3 percent) after 11 years in the compliant patients and 48.9 percent (18.8 and 79.0 percent) after 9 years in the noncompliant patients (P less than 0.0001). A total of 110 patients had overt nephropathy when they started to take colchicine. Among 86 patients who had proteinuria but not the nephrotic syndrome, proteinuria resolved in 5 and stabilized in 68 (for more than eight years in 40). Renal function deteriorated in 13 of the patients with proteinuria and in all of the 24 patients with the nephrotic syndrome or uremia. We conclude that colchicine prevented amyloidosis in our high-risk population and that it can prevent additional deterioration of renal function in patients with amyloidosis who have proteinuria but not the nephrotic syndrome.
为了确定秋水仙碱能否预防或改善家族性地中海热患者的淀粉样变性,我们对1070例家族性地中海热患者进行了随访,这些患者在被建议服用秋水仙碱以预防发热性发作后,随访时间为4至11年。总体而言,在研究结束时,肾病的患病率是在秋水仙碱用于治疗家族性地中海热之前进行的一项研究中的三分之一。在最初没有淀粉样变性证据的960例患者中,坚持预防方案的4例出现蛋白尿,而在54例承认未坚持方案的患者中有16例出现蛋白尿。生命表分析显示,坚持方案的患者11年后蛋白尿累积发生率为1.7%(90%置信区间为0.0至11.3%),未坚持方案的患者9年后蛋白尿累积发生率为48.9%(18.8至79.0%)(P<0.0001)。共有110例患者开始服用秋水仙碱时已有明显肾病。在86例有蛋白尿但无肾病综合征的患者中,5例蛋白尿消失,68例蛋白尿稳定(40例稳定超过8年)。13例有蛋白尿的患者以及所有24例有肾病综合征或尿毒症的患者肾功能恶化。我们得出结论,秋水仙碱在我们的高危人群中预防了淀粉样变性,并且它可以预防有蛋白尿但无肾病综合征的淀粉样变性患者肾功能的进一步恶化。