Amsterdam UMC, Location Vrije Universiteit Amsterdam, Medical Psychology, Amsterdam, The Netherlands.
Amsterdam Public Health, Mental Health, Amsterdam, The Netherlands.
Diabet Med. 2024 Sep;41(9):e15385. doi: 10.1111/dme.15385. Epub 2024 Jun 14.
Recently, efforts have been made to use and report person-reported outcomes (PROs) in randomised clinical trials (RCTs). Here, we aim to (1) assess the status of inclusion of PROs in registered RCTs over 5 years in people with type 1 or 2 diabetes, and (2) map the PRO measures (PROMs) onto predefined domains.
The largest trial registries (Clinicatrials.gov, International Clinical Trial Platform and ISRCTN) were systematically searched for RCTs in people with type 1 and/or type 2 diabetes of all ages between 2018 and 2023. Coding of PROs comprised: (1) PRO measure(s) included yes or no; if yes: (2) PRO(s) as primary outcome yes or no; and (3) mapping PROMs onto predefined PRO domains and per type of intervention.
N = 1543 trials met our inclusion criteria, of which n = 673 (44%) included PROs, assessed by 545 different measures. Twenty per cent of drug trials (n = 112) and 71% of behavioural interventions (n = 405) included PROs. In 149 trials (9.6%), a PRO was the primary outcome. The psychological functioning domain was most often assessed across all trials (21.6%), specifically in behavioural (44.8%) and medical device interventions (29.7%). In drug trials, the physical functioning and functional health domain was most included (9%). Across all trials, the social and family functioning domain was least assessed (3%).
We noticed an increase in the inclusion of PROs in diabetes RCTs. However, PROs are rarely included as primary outcomes in the majority of studies, particularly in drug trials. The heterogeneity of PROMs used in RCTs underscores the need for standardisation of PROs.
最近，人们努力在随机临床试验（RCT）中使用和报告患者报告的结局（PROs）。在此，我们旨在：（1）评估在 5 年内，1 型或 2 型糖尿病患者的注册 RCT 中纳入 PRO 的情况；（2）将 PRO 测量指标（PROMs）映射到预定义的领域。
系统检索了最大的试验注册处（Clinicatrials.gov、国际临床试验平台和 ISRCTN）中所有年龄段的 1 型和/或 2 型糖尿病患者的 RCT。PRO 的编码包括：（1）是否纳入 PRO 测量指标；如果是：（2）PRO 作为主要结局的评估；（3）将 PROM 映射到预定义的 PRO 领域以及每种干预类型。
符合纳入标准的 RCT 共有 1543 项，其中 673 项（44%）纳入了 PRO，采用了 545 种不同的测量方法。20%的药物试验（n=112）和 71%的行为干预试验（n=405）纳入了 PRO。在 149 项试验（9.6%）中，PRO 是主要结局。在所有试验中，心理功能领域的评估最为常见（21.6%），尤其是在行为（44.8%）和医疗器械干预（29.7%）中。在药物试验中，身体功能和功能健康领域的纳入最为常见（9%）。在所有试验中，社会和家庭功能领域的评估最少（3%）。
我们注意到在糖尿病 RCT 中纳入 PRO 的情况有所增加。然而，在大多数研究中，PRO 很少被作为主要结局纳入，尤其是在药物试验中。RCT 中使用的 PROMs 存在异质性，这突显了 PRO 标准化的必要性。
