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秋水仙碱治疗家族性地中海热淀粉样肾病的疗效

Efficacy of colchicine therapy in amyloid nephropathy of familial Mediterranean fever.

作者信息

Oner Ayşe, Erdoğan Ozlem, Demircin Gülay, Bülbül Mehmet, Memiş Leyla

机构信息

Department of Pediatric Nephrology, Dr. Sami Ulus Children's Hospital, Ankara, Turkey.

出版信息

Pediatr Nephrol. 2003 Jun;18(6):521-6. doi: 10.1007/s00467-003-1129-x. Epub 2003 Apr 16.

Abstract

The aim of this study was to investigate the effect of colchicine therapy on the outcome of amyloid nephropathy of familial Mediterranean fever (FMF) in childhood. The diagnosis of amyloidosis type AA was confirmed by renal biopsy in 38 patients. During a mean follow-up period of 30.5 months (range 6-88 months), the patients received colchicine therapy. While 24 of these patients were compliant with the treatment, 14 patients remained non-compliant. Of the 24 compliant patients, 19 had normal renal function at the onset; in 13 the proteinuria improved, in 5 patients it remained stable, and in 1 patient it deteriorated from a proteinuric to nephrotic stage. Partial resolution of amyloidosis was demonstrated by repeat renal biopsy in 1 patient who showed complete resolution of proteinuria. In contrast, none of 14 non-compliant patients improved, and while only 1 patient was in renal failure initially, 10 patients deteriorated to renal failure during the follow-up period. The presence of tubulointerstitial injury at presentation adversely affected the prognosis. In conclusion, when used appropriately, colchicine can improve proteinuria and prevent chronic renal failure in patients with amyloid nephropathy of FMF. The presence of renal failure or tubulointerstitial injury at presentation and non-compliance with therapy are the factors decreasing the success of therapy.

摘要

本研究的目的是调查秋水仙碱治疗对儿童家族性地中海热(FMF)淀粉样肾病预后的影响。通过肾活检确诊38例AA型淀粉样变性患者。在平均30.5个月(范围6 - 88个月)的随访期内,患者接受秋水仙碱治疗。其中24例患者依从治疗,14例患者仍不依从。在24例依从治疗的患者中,19例起病时肾功能正常;13例蛋白尿改善,5例稳定,1例从蛋白尿期恶化为肾病期。1例经重复肾活检显示蛋白尿完全缓解,证实淀粉样变性部分消退。相比之下,14例不依从治疗的患者均无改善,虽然最初只有1例患者出现肾衰竭,但随访期间有10例患者恶化为肾衰竭。就诊时存在肾小管间质损伤对预后产生不利影响。总之,适当使用时,秋水仙碱可改善FMF淀粉样肾病患者的蛋白尿并预防慢性肾衰竭。就诊时存在肾衰竭或肾小管间质损伤以及不依从治疗是降低治疗成功率的因素。

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