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肾小球疾病:为何高质量临床试验如此匮乏?

Glomerular disease: why is there a dearth of high quality clinical trials?

机构信息

Department of Medicine, Columbia University College of Physicians and Surgeons, New York, New York, USA.

出版信息

Kidney Int. 2010 Aug;78(4):337-42. doi: 10.1038/ki.2010.156. Epub 2010 Jun 2.

Abstract

There is a paucity of high quality clinical trials in glomerular disease, particularly in non-diabetic kidney disease. The aims of this review include quantifying the extent of this problem and exploring reasons for the scarcity of such trials in primary glomerular disease, with an emphasis on immunoglobulin A nephropathy, minimal change disease, focal segmental glomerulosclerosis, and membranous nephropathy in comparison with the more common diseases of diabetic nephropathy and lupus nephritis. Reasons for the dearth of high quality clinical trials in primary glomerular disease include (1) low prevalence of disease; (2) variability in clinical presentation; (3) variability in treatment response; (4) lack of consensus in definitions; (5) difficulty in recruiting patients; (6) high costs of randomized controlled trials; and (7) lack of collaborative efforts. To facilitate greater numbers of high quality clinical trials in glomerular disease, practice guidelines should establish common classification systems of disease and common clinical end points, industry and non-industry sponsored research should find common ground and work together toward advancing science, and national registries should be created to encourage collaborations across institutions and across nations.

摘要

肾小球疾病的高质量临床试验相对较少,特别是在非糖尿病肾病中。本综述的目的包括定量评估这一问题的严重程度,并探讨原发性肾小球疾病中此类试验稀缺的原因,重点关注 IgA 肾病、微小病变性肾病、局灶节段性肾小球硬化和膜性肾病,与更常见的糖尿病肾病和狼疮性肾炎进行比较。原发性肾小球疾病中高质量临床试验稀缺的原因包括:(1)疾病的低患病率;(2)临床表现的变异性;(3)治疗反应的变异性;(4)定义缺乏共识;(5)患者招募困难;(6)随机对照试验的高成本;以及(7)缺乏协作努力。为了促进肾小球疾病中更多高质量临床试验的开展,实践指南应制定共同的疾病分类系统和共同的临床终点,行业和非行业赞助的研究应找到共同点,共同努力推进科学,国家登记处应建立,以鼓励机构间和国家间的合作。

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