Blair Jo, Gregory John W, Hughes Dyfrig, Ridyard Colin H, Gamble Carrol, McKay Andrew, Didi Mohammed, Thornborough Keith, Bedson Emma, Awoyale Lola, Cwiklinski Emma, Peak Matthew
Department of Endocrinology, Alder Hey Children's NHS Foundation Trust, Liverpool, L12 2AP, UK.
Department of Child Health, School of Medicine, Cardiff University, Cardiff, CF14 4XN, Wales, UK.
Trials. 2015 Apr 16;16:163. doi: 10.1186/s13063-015-0658-5.
Intensive insulin therapy with continuous subcutaneous insulin infusion (CSII) devices or multiple daily injections (MDI) reduces the risk of long-term vascular complications of type I diabetes (TID). Both treatments are used routinely, but there is little evidence to demonstrate superiority of either treatment. If CSII treatment reduces the risk of long-term complications or is associated with an improved quality of life (QoL), the additional cost of this therapy may be compensated for by a reduction in long-term health expenditure. If there is no demonstrable difference between treatments, health-care resources may be better invested elsewhere. This study aims to address this gap in knowledge.
METHODS/DESIGN: This is a pragmatic, randomised controlled trial (RCT). Fifteen centres, selected to represent a population with a broad demographic, will recruit 316 patients, newly diagnosed with TID, aged between 7 months and 15 years. Exclusion criteria include additional pathologies or treatments likely to affect glycaemic control and a first-degree relative with TID. Randomisation to CSII or MDI is stratified for age, gender and recruiting centre. The randomised treatment starts within 15 days of diagnosis. Patients will be trained to adjust their insulin dose according to carbohydrate intake and blood glucose level. Study visits coincide with routine clinic appointments at 3, 6, 9 and 12 months when data relating to routine clinical assessments, adverse events and concomitant medications are collected. Health utilities questionnaires are completed at each visit and a diabetes-specific QoL questionnaire (PedsQL) at diagnosis, 6 and 12 months. The primary outcome is glycaemic control (HbA1c) at 12 months. Secondary outcome measures include QoL, insulin use, growth and weight gain, adverse events and a health economics appraisal.
This is the first adequately powered RCT comparing CSII and MDI in a non-selected population, treated according to standard practice guidelines. It will produce data that are meaningful to individual patients and local and national policymakers.
The study was registered with the European Clinical Trials Database on 4 November 2010, reference 2010-023792-25.
采用持续皮下胰岛素输注（CSII）装置或每日多次注射（MDI）进行强化胰岛素治疗可降低1型糖尿病（TID）长期血管并发症的风险。这两种治疗方法均常规使用，但几乎没有证据表明哪种治疗方法更具优势。如果CSII治疗能降低长期并发症的风险或改善生活质量（QoL），那么这种治疗方法的额外费用可能会因长期医疗支出的减少而得到补偿。如果两种治疗方法之间没有明显差异，那么医疗资源可能更好地投资于其他地方。本研究旨在填补这一知识空白。
方法/设计：这是一项实用的随机对照试验（RCT）。选取15个中心，代表广泛的人口统计学特征，将招募316例新诊断的TID患者，年龄在7个月至15岁之间。排除标准包括可能影响血糖控制的其他病理状况或治疗方法以及患有TID的一级亲属。根据年龄、性别和招募中心对CSII或MDI进行随机分组。随机治疗在诊断后15天内开始。患者将接受培训，根据碳水化合物摄入量和血糖水平调整胰岛素剂量。研究访视与3、6、9和12个月的常规门诊预约时间一致，届时收集与常规临床评估、不良事件和伴随用药相关的数据。每次访视时完成健康效用问卷，并在诊断时、6个月和12个月时完成特定于糖尿病的生活质量问卷（儿童生活质量量表）。主要结局是12个月时的血糖控制（糖化血红蛋白）。次要结局指标包括生活质量、胰岛素使用情况、生长和体重增加、不良事件以及健康经济学评估。
这是第一项在未选择的人群中，按照标准实践指南比较CSII和MDI的有足够效力的RCT。它将产生对个体患者以及地方和国家政策制定者有意义的数据。
该研究于2010年11月4日在欧洲临床试验数据库注册，注册号为2010 - 023792 - 25。
