The long-term effects of insulin use in incident cystic fibrosis-related diabetes: a target trial emulated using longitudinal national registry data
The long-term effects of insulin use in incident cystic fibrosis-related diabetes: a target trial emulated using longitudinal national registry data
复制标题
DOI：
10.1183/23120541.00170-2022
复制
发表时间：
2022-10-01
影响因子：
4.6
通讯作者：
Frost, Freddy
中科院分区：
文献类型：
--
作者：
Granger, Emily;Keogh, Ruth H.;Frost, Freddy
囊性纤维化相关性糖尿病（CFRD）是囊性纤维化的常见并发症，与有害的临床结果相关。国际指南推荐使用胰岛素进行治疗。然而，很少有临床试验数据支持胰岛素的使用，而且对治疗的长期结果知之甚少。本研究的目的是比较CFRD中使用胰岛素与不使用胰岛素的长期影响。方法：我们使用来自英国国家囊性纤维化登记处的数据，并采用目标试验框架。符合条件的个体包括新诊断为CFRD的12岁及以上的患者。结果是在5年随访期间预测1秒内用力呼气量（FEV1 %）和体重指数z分数（BMI）的变化。治疗策略是在随访期间接受胰岛素或不接受胰岛素。采用两种方法获得治疗效果估计，以控制混杂：边际结构模型的处理逆概率加权估计和g公式。我们在2008年至2016年期间确定了1613例诊断为CFRD的个体，其中1196例和1192例分别纳入了fev1%和BMI结果分析。在5年的研究期间，我们没有发现胰岛素对FEV1 %有影响的证据。同样，我们没有发现胰岛素对BMI的总体影响；然而，有证据表明对基线BMI较低的患者有积极的治疗效果。结论：使用完善的国家登记数据，我们没有发现胰岛素对CFRD患者FEV1或BMI的长期治疗效果的证据。
Introduction Cystic fibrosis-related diabetes (CFRD) is a common complication of cystic fibrosis and is associated with deleterious clinical outcomes. Insulin is recommended as a treatment by international guidelines. However, there are scarce clinical trial data to support the use of insulin, and little is known about the long-term outcomes of treatment. The aim of this study was to compare the long-term impacts of insulin use versus non-use in CFRD.Methods We used data from the national UK Cystic Fibrosis Registry and adopted a target trial framework. Eligible individuals included those 12 years and older with a new diagnosis of CFRD. Outcomes were change in % predicted forced expiratory volume in 1 s (FEV1 %) and body mass index z-scores (BMI) over a 5-year follow-up period. Treatment strategies were to receive insulin or not for the duration of follow-up. Treatment effect estimates were obtained using two methods to control for confounding: inverse-probability-of-treatment weighted estimation of marginal structural models and the G-formula.Results We identified 1613 individuals diagnosed with CFRD between 2008 and 2016 and included 1196 and 1192 in the FEV1 % and BMI outcome analyses respectively. We found no evidence of an effect of insulin on FEV1 % over the 5-year study period. Similarly, we found no overall effect of insulin on BMI; however, there was some evidence for a positive treatment effect in patients with lower baseline BMI.Conclusion Using well-established national registry data, we found no evidence of long-term treatment effects for insulin on FEV1 % or BMI in people with incident CFRD.