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

共 50 条

[21] Advocacy for persons with rare diseases: a strategy for creating influence
Christopher C Chang
Victoria Gall
Matthew H Liang
Nature Clinical Practice Rheumatology, 2007, 3 : 421 - 421
[22] A registry for rare benign diseases - A multicenter approach in Germany
Micke, O
Seegenschmiedt, MH
Hesselmann, S
Makoski, HB
STRAHLENTHERAPIE UND ONKOLOGIE, 2002, 178 : 99 - 99
[23] Reporting on the unkown: A registry for rare benign diseases in Germany
Micke, O.
Seegenschmiedt, M. H.
Makoski, H.-B.
STRAHLENTHERAPIE UND ONKOLOGIE, 2007, 183 : 147 - 147
[24] National platform for Rare Diseases Data Registry of Japan
Furusawa, Yoshihiko
Yamaguchi, Izumi
Yagishita, Naoko
Tanzawa, Kazumasa
Matsuda, Fumihiko
Yamano, Yoshihisa
Yamada, Ryo
Tabara, Yasuharu
Kamatani, Yoichiro
Kawaguchi, Syuji
Kosugi, Shinji
Higasa, Koichiro
Matsubara, Yoichi
Matsumoto, Naomichi
Aasano, Yoshihiro
Nishino, Ichizo
Nakamura, Harumasa
Takano, Atsushi
Iot, Motoi
Sakai, Shinya
LEARNING HEALTH SYSTEMS, 2019, 3 (03):
[25] A first description of the Colombian national registry for rare diseases
Mateus H.E.
Pérez A.M.
Mesa M.L.
Escobar G.
Gálvez J.M.
Montaño J.I.
Ospina M.L.
Laissue P.
BMC Research Notes, 10 (1)
[26] Where there is no evidence: A registry for rare benign diseases in Germany
Micke, O.
Seegenschmiedt, M. H.
Hesselmann, S.
Makoski, H. -B
STRAHLENTHERAPIE UND ONKOLOGIE, 2006, 182 : 68 - 69
[27] A registry for rare benign diseases - A multicenter approach in Germany
Micke, O
Seegenschmiedt, MH
RADIOTHERAPY AND ONCOLOGY, 2004, 71 : S43 - S43
[28] Clinical Trials and Rare Diseases
Gerss, Joachim Werner Otto
Koepcke, Wolfgang
RARE DISEASES EPIDEMIOLOGY, 2010, 686 : 173 - 190
[29] Clinical trials and rare diseases
Lagakos, SW
NEW ENGLAND JOURNAL OF MEDICINE, 2003, 348 (24): : 2455 - 2456
[30] Creating an Innovation Engine to Advance Medicine for Patients with Rare Diseases
Gallagher, Thomas
HUMAN GENE THERAPY, 2025, 36 (1-2) : 1 - 6

← 1 2 3 4 5 →