Defining Pathways for Development of Disease-Modifying Therapies in Children With Type 1 Diabetes: A Consensus Report

被引：63

作者：

Wherrett, Diane K. ^{[1
]}

Chiang, Jane L. ^{[2
]}

Delamater, Alan M. ^{[3
]}

DiMeglio, Linda A. ^{[4
]}

Gitelman, Stephen E. ^{[5
]}

Gottlieb, Peter A. ^{[6
,7
]}

Herold, Kevan C. ^{[8
]}

Lovell, Daniel J. ^{[9
]}

Orchard, Trevor J. ^{[10
]}

Ryan, Christopher M. ^{[11
]}

Schatz, Desmond A. ^{[12
]}

Wendler, David S. ^{[13
]}

Greenbaum, Carla J. ^{[14
]}

机构：

[1] Univ Toronto, Dept Pediat, Toronto, ON, Canada

[2] Amer Diabet Assoc, Alexandria, VA USA

[3] Univ Miami, Dept Pediat, Miami, FL 33152 USA

[4] Indiana Univ, Dept Pediat, Indianapolis, IN 46204 USA

[5] Univ Calif San Francisco, Dept Pediat, San Francisco, CA USA

[6] Univ Colorado, Dept Med, Denver, CO USA

[7] Univ Colorado, Dept Pediat, Denver, CO 80202 USA

[8] Yale Univ, Dept Immunobiol, New Haven, CT USA

[9] Cincinnati Childrens Hosp Med Ctr, Div Rheumatol, Cincinnati, OH 45229 USA

[10] Univ Pittsburgh, Grad Sch Publ Hlth, Pittsburgh, PA USA

[11] Univ Pittsburgh, Dept Psychiat, Pittsburgh, PA USA

[12] Univ Florida, Dept Pediat, Gainesville, FL USA

[13] NIH, Dept Bioeth, Bethesda, MD 20892 USA

[14] Benaroya Res Inst, Diabet Res Program, Seattle, WA 98101 USA

来源：

DIABETES CARE | 2015年 / 38卷 / 10期

关键词：

QUALITY-OF-LIFE; RANDOMIZED CONTROLLED-TRIAL; PRESERVES C-PEPTIDE; BETA-CELL FUNCTION; PITTSBURGH EPIDEMIOLOGY; DOUBLE-BLIND; PEDIATRIC RESEARCH; GLYCEMIC CONTROL; RISK-FACTORS; NATURAL-HISTORY;

D O I：

10.2337/dc15-1429

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Emerging data suggest that type 1 diabetes is a more aggressive disease in children than in adults, with important differences in pathophysiology and clinical course. Therefore, the efficacy of disease-modifying therapies may be different in the two populations. Understanding the developmental and regulatory pathways for type 1 diabetes-modifying therapies in children will enable industry, academia, funders, advocacy groups, and regulators to translate new science to clinical care. This consensus report characterizes the fundamental differences in type 1 diabetes between children and adults and proposes a thoughtful approach to better understand the development and regulatory pathways for type 1 diabetes therapies.

引用

页码：1975 / 1985

页数：11

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