在1型糖尿病发病前进行干预：欧洲烟酰胺糖尿病干预试验（ENDIT）的基线数据。
Intervening before the onset of Type 1 diabetes: baseline data from the European Nicotinamide Diabetes Intervention Trial (ENDIT).
出版信息
Diabetologia. 2003 Mar;46(3):339-46. doi: 10.1007/s00125-003-1033-8. Epub 2003 Feb 27.
AIMS/HYPOTHESIS: To set up a clinical trial to establish whether nicotinamide can prevent or delay clinical onset of Type 1 diabetes.
METHOD
The European Nicotinamide Diabetes Intervention Trial is a randomised, double-blind, placebo-controlled intervention trial undertaken in 18 European countries, Canada and the USA. Entry criteria were a first-degree family history of Type 1 diabetes, age 3-40 years, confirmed islet cell antibody (ICA) levels greater than or equal to 20 JDF units, and a non-diabetic OGTT; the study group was further characterised by intravenous glucose tolerance testing, measurement of antibodies to GAD, IA-2 and insulin and HLA class II genotyping.
RESULTS
ICA screening was carried out in approximately 30,000 first-degree relatives. A total of 1004 individuals fulfilled ICA criteria for eligibility, and 552 (288 male) were randomised to treatment. Of these, 331 were aged less than 20 years (87% siblings and 13% offspring of the proband with diabetes) and 221 were 20 years of age or more (76% parents, 21% siblings and 3% offspring). Oral glucose tolerance was normal in 500 and impaired in 52 (9.4%), and first phase insulin response in the IVGTT was below the 10(th) centile in 34%. Additional islet autoantibodies were identified in 354 trial entrants. Diabetes-associated HLA class II haplotypes were found in 84% of the younger age group and 80% of the older group. The protective haplotype HLA-DQA10102-DQB10602 was found in 10% overall.
CONCLUSIONS/INTERPRETATION: ENDIT has shown that a trial of an intervention designed to halt or delay progression to Type 1 diabetes can be carried out on a multinational collaborative basis, as and when potentially safe and effective forms of intervention become available. Primary screening with biochemically defined autoantibodies will substantially reduce the number of lower risk individuals to be included in future intervention trials
目的/假设：开展一项临床试验，以确定烟酰胺是否能够预防或延缓1型糖尿病的临床发病。
方法
欧洲烟酰胺糖尿病干预试验是一项在18个欧洲国家、加拿大和美国进行的随机、双盲、安慰剂对照干预试验。入选标准为1型糖尿病一级家族史、年龄3至40岁、确诊的胰岛细胞抗体（ICA）水平大于或等于20 JDF单位以及非糖尿病口服葡萄糖耐量试验（OGTT）；研究组进一步通过静脉葡萄糖耐量试验、谷氨酸脱羧酶（GAD）抗体、胰岛抗原2（IA-2）抗体和胰岛素抗体检测以及人类白细胞抗原（HLA）II类基因分型进行特征分析。
结果
对约30000名一级亲属进行了ICA筛查。共有1004人符合ICA入选标准，其中552人（288名男性）被随机分配接受治疗。其中，331人年龄小于20岁（87%为糖尿病先证者的兄弟姐妹，13%为后代），221人年龄在20岁及以上（76%为父母，21%为兄弟姐妹，3%为后代）。500人口服葡萄糖耐量正常，52人（9.4%）受损，静脉葡萄糖耐量试验中第一阶段胰岛素反应低于第10百分位数的占34%。354名试验参与者检测出其他胰岛自身抗体。糖尿病相关的HLA II类单倍型在较年轻组中占84%，在较年长组中占80%。总体上，保护性单倍型HLA-DQA10102-DQB10602占10%。
结论/解读：欧洲烟酰胺糖尿病干预试验表明，当有潜在安全有效的干预形式时，针对阻止或延缓进展为1型糖尿病的干预试验可以在跨国合作的基础上开展。使用生化定义的自身抗体进行初步筛查将大幅减少未来干预试验中纳入的低风险个体数量。
Zotero 插件