Relationship between participant-reported outcomes, residual beta cell function and metabolic parameters in youth with newly diagnosed type 1 diabetes.

However, the expected changes in PROMs scores immediately following diagnosis and their association with residual beta cell function, metabolic markers and continuous glucose monitoring (CGM) are unclear.

然而，诊断后PROMs得分的预期变化及其与残余β细胞功能、代谢标志物和连续葡萄糖监测（CGM）的关联尚不清楚。

Methods

Repeated PROMs including Paediatric Quality of Life Inventory diabetes module (PedsQL) and hypoglycaemia fear survey (HFS) were recorded from participants aged 10-18 years with newly diagnosed type 1 diabetes and their parents in two clinical trials: CLOuD (N=97, hybrid closed loop [HCL] vs multiple daily injections [MDI]) and USTEKID (N=72, ustekinumab immunotherapy vs placebo).

在两项临床试验中记录了重复的PROMs，包括10-18岁新诊断为1型糖尿病的参与者及其父母的儿童生活质量清单糖尿病模块（PedsQL）和低血糖恐惧调查（HFS）：CLOuD（N=97，混合闭环[HCL]与多次日注射[MDI]）和USTEKID（N=72，乌司他木单抗免疫治疗与安慰剂）。

Scores were compared with serial mixed meal-stimulated C-peptide levels (AUC C-peptide), HbA1c and CGM data.

得分与序贯混合餐刺激C肽水平（AUC C肽）、HbA1c和CGM数据进行了比较。

Results

PedsQL and HFS scores for children/adolescents and their parents showed wide variation between individuals but did not change substantially within individuals over the first 48 months from diagnosis.

儿童/青少年及其父母的PedsQL和HFS得分在个体间显示出很大的差异，但在诊断后的前48个月内个体内的变化不大。

Baseline scores were highly predictive of scores at 12-48 months (p<0.001).

基线得分对12-48个月的得分具有很高的预测性（p<0.001）。

PedsQL scores were higher (better) in those reported by children/adolescents than by their parents (p<0.01).

儿童/青少年报告的PedsQL得分高于（更好）其父母报告的得分（p<0.01）。

In contrast, HFS scores were higher in parents than children (p<0.001), indicating more fear.

相比之下，父母的HFS得分高于儿童（p<0.001），表明更多的恐惧。

Strong correlations were observed between child and parent scores (p<0.001).

观察到儿童和父母评分之间存在强相关性（p<0.001）。

No significant improvement in these scores was detected following intervention (ustekinumab or HCL).

干预后（使用乌司他木单抗或HCL），这些评分没有显著改善。

Meta-analysisrevealed modest but statistically significant associations between HbA1c and PedsQL (β(std)=-0.11; 95% CI -0.20, -0.03) and HFS (β(std)=0.11; 95% CI 0.00, 0.21), and between CGM time in range and PedsQL (β(std)=0.14; 95% CI 0.03, 0.26) but not HFS (β(std)=-0.05; 95% CI -0.16, 0.06).

荟萃分析揭示了HbA1c与PedsQL（β(std)=-0.11; 95% CI -0.20, -0.03）和HFS（β(std)=0.11; 95% CI 0.00, 0.21）之间，以及CGM时间在范围内与PedsQL（β(std)=0.14; 95% CI 0.03, 0.26）之间存在适度但统计学上显著的关联，但与HFS（β(std)=-0.05; 95% CI -0.16, 0.06）之间没有显著关联。

Beta cell function (AUC C-peptide) was strongly associated with HbA1c (β(std)=-0.29; 95% CI -0.39, -0.20) and CGM time in range (β(std)=0.41; 95% CI 0.30, 0.52).

β细胞功能（AUC C肽）与HbA1c（β(std)=-0.29; 95% CI -0.39, -0.20）和CGM时间在范围内（β(std)=0.41; 95% CI 0.30, 0.52）有很强的关联。

Higher beta cell function showed a trend towards better PedsQL (β(std)=0.11; 95% CI -0.03, 0.25) and lower HFS (β(std)=-0.05; 95% CI -0.17, 0.07) but this did not reach statistical significance.

较高的β细胞功能显示出趋向于更好的PedsQL（β(std)=0.11; 95% CI -0.03, 0.25）和较低的HFS（β(std)=-0.05; 95% CI -0.17, 0.07），但这并未达到统计学显著性。

conclusions/interpretation

PedsQL and HFS scores changed little during the first 48 months after diagnosis of type 1 diabetes.

在诊断为1型糖尿病后的前48个月内，PedsQL和HFS评分变化不大。

These scores showed modest but statistically significant associations with measures of glucose management (HbA1c and CGM time in range), whereas the relationships with residual beta cell function (C-peptide) were weaker and did not reach significance.

这些评分与血糖管理的测量（HbA1c和CGM时间在范围内）显示出适度但统计学上显著的关联，而与残余β细胞功能（C肽）的关系较弱，且未达到显著性。

The modest size of these effects suggests current PROMs capture only limited aspects of the clinical benefit associated with beta cell preservation.

这些效应的适度大小表明，当前的PROMs仅捕获了与β细胞保护相关的临床益处的有限方面。

Future research should incorporate psychometric instruments that are specifically adapted for young people using modern diabetes technologies and undergoing disease-modifying therapy, to ensure outcomes are meaningfully represented in early-stage type 1 diabetes trials.

未来的研究应纳入专门为使用现代糖尿病技术和接受疾病改变治疗的年轻人量身定制的心理测量工具，以确保在早期1型糖尿病试验中结果得到有意义的体现。