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罕见病因所致肾衰竭患者的终末期肾病结局。I. 遗传性和代谢性疾病。

Outcome of end-stage renal disease in patients with rare causes of renal failure. I. Inherited and metabolic disorders.

作者信息

Nissenson A R, Port F K

机构信息

UCLA School of Medicine 90024.

出版信息

Q J Med. 1989 Nov;73(271):1055-62.

PMID:2516341
Abstract

This study represents the first national multi-center evaluation of treatment for end-stage renal disease outcome in patients with rare metabolic and inherited disorders in the United States. Because of the small number of such patients at single centers only a co-operative study could provide adequate data on enough patients for meaningful conclusions. By co-ordinating such data, the Network Forum provides a model for the newly organized U.S. Renal Data System and demonstrates the potential for useful special studies in the future. It is clear from these data that despite the systemic nature of these illnesses, treatment for ESRD can be applied to achieve excellent survival rates.

摘要

本研究是美国首次针对患有罕见代谢和遗传性疾病的终末期肾病患者治疗效果进行的全国性多中心评估。由于单中心此类患者数量较少,只有合作研究才能为足够多的患者提供足够的数据以得出有意义的结论。通过协调此类数据,网络论坛为新组建的美国肾脏数据系统提供了一个模式,并展示了未来开展有用的专项研究的潜力。从这些数据可以清楚地看出,尽管这些疾病具有全身性,但可以应用终末期肾病治疗方法来实现极高的存活率。

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