Creating an effective clinical registry for rare diseases

被引：33

作者：

D'Agnolo, Hedwig M. A. ^{[1
]}

Kievit, Wietske ^{[2
]}

Andrade, Raul J. ^{[3
,4
,5
]}

Karlsen, Tom Hemming ^{[6
]}

Wedemeyer, Heiner ^{[7
]}

Drenth, Joost P. H. ^{[1
]}

机构：

[1] Radboud Univ Nijmegen, Med Ctr, Dept Gastroenterol & Hepatol, POB 9101, NL-6500 HB Nijmegen, Netherlands

[2] Radboud Univ Nijmegen, Med Ctr, Radboud Inst Hlth Sci, NL-6525 ED Nijmegen, Netherlands

[3] Virgin Victory Univ Hosp, Inst Biomed Res Malaga IBIMA, Malaga, Spain

[4] Univ Malaga, Sch Med, Malaga, Spain

[5] Univ Malaga, Hosp Univ Virgen Victoria, Inst Invest Biomed Malaga, Unidad Gest Clin Aparato Digest,Ctr Invest Biomed, E-29071 Malaga, Spain

[6] Oslo Univ Hosp, Dept Transplantat Med, Div Canc Med Surg & Transplantat, Oslo, Norway

[7] Hannover Med Sch, Dept Gastroenterol Hepatol & Endocrinol, Hannover, Germany

来源：

UNITED EUROPEAN GASTROENTEROLOGY JOURNAL | 2016年 / 4卷 / 03期

关键词：

Clinical registry; database; gastrointestinal disease; liver disease; liver transplantation; practical guide; rare diseases; rare liver disorders; registry design; PRIMARY BILIARY-CIRRHOSIS; REPORTED OUTCOME MEASURES; HEPATOCELLULAR-CARCINOMA; EPIDEMIOLOGY; MANAGEMENT; RISK;

D O I：

10.1177/2050640615618042

中图分类号：

R57 [消化系及腹部疾病];

学科分类号：

摘要：

The exposure of clinicians to patients with rare gastrointestinal diseases is limited. This hurts clinical studies, which impedes accumulation of scientific knowledge on the natural disease course, treatment outcomes and prognosis in these patients. An excellent method to detect patterns on an aggregate level that would not be possible to discover in individual cases, is a registry study. This paper aims to describe a template to create a successful international registry for rare diseases. We focus mainly on rare hepatic diseases, but lessons from this paper serve other fields in medicine, as well.

引用

页码：333 / 338

页数：6

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