WS16.03The prevalence and probability of cystic fibrosis-related diabetes in children under 10: a prospective cohort study

IF 5.4 2区医学 Q1 RESPIRATORY SYSTEM

Journal of Cystic Fibrosis Pub Date : 2025-06-01 DOI:10.1016/j.jcf.2025.03.584

S. Kiss , K. Ocskay , P. Mátrai , A. Párniczky

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引用次数: 0

Abstract

Objectives

With the widespread adoption of cystic fibrosis transmembrane conductance modulator (CFTRm) therapies, the metabolic profile of people with cystic fibrosis is changing. We aimed to assess the prevalence and probability of the development of cystic fibrosis-related diabetes (CFRD) under the age of 10.

Methods

Data from the prospective CF-related Pancreatic Disorders Registry was analyzed using time-to-event analysis, the event being the diagnosis of CFRD. Kaplan-Meier curves illustrated the event as a function of age, for the total cohort and by sex as well. Log-rank tests compared males and females. Age at diagnosis was analyzed using histograms, calculating mean, median, and the percentage diagnosed under the age of 10. Eleven patients were excluded from time-to-event analyses who presented with CFRD at their first visit.

Results

Of the 135 children in the cohort (Table 1), 31 were diagnosed with CFRD, 11 at the beginning of follow-up and 20 during. Of all CFRD patients, 48.4% (n=15) were diagnosed before the age of 10 (Table 2), including 5 (16.1%) from those having CFRD at the beginning of follow-up. The analysis of patients without pre-existing CFRD indicated a 13.2% (95% CI: [5.1%; 20.5%]) probability of CFRD by the age of 10 years. No significant sex-based differences were observed in CFRD risk (p=0.353).

Conclusion

The observed distribution of CFRD diagnosis suggests a potential need for earlier screening in a subset of patients. Ongoing analyses incorporating modulator therapy as a covariate will provide a more complete understanding and inform optimal screening protocols.

Funding: National Research, Development and Innovation Fund (NRDI Fund) FK 138929; CF Trust Strategic Research Consortium Grant NU-000600

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10岁以下儿童囊性纤维化相关糖尿病的患病率和概率：一项前瞻性队列研究

随着囊性纤维化跨膜传导调节剂（CFTRm）治疗的广泛采用，囊性纤维化患者的代谢谱正在发生变化。我们的目的是评估10岁以下囊性纤维化相关糖尿病（CFRD）的患病率和发展概率。方法对前瞻性cf相关胰腺疾病登记处的数据进行时间-事件分析，事件为CFRD的诊断。Kaplan-Meier曲线说明了该事件是年龄的函数，对于整个队列和性别也是如此。Log-rank检验比较了雄性和雌性。使用直方图分析诊断年龄，计算平均值、中位数和10岁以下诊断的百分比。11例首次就诊时出现CFRD的患者被排除在事件时间分析之外。结果在该队列的135名儿童中（表1），31名被诊断为CFRD， 11名在随访开始时，20名在随访期间。在所有CFRD患者中，48.4% （n=15）在10岁之前被诊断出来（表2），其中5例（16.1%）来自随访开始时已患有CFRD的患者。对未存在CFRD的患者的分析表明：13.2% (95% CI: [5.1%；（20.5%）) 10岁时发生CFRD的概率。在CFRD风险方面，未观察到显著的性别差异（p=0.353）。结论观察到的CFRD诊断分布提示有可能需要对一部分患者进行早期筛查。正在进行的分析将调节剂治疗作为协变量，将提供更完整的理解，并告知最佳筛选方案。资助：国家研究、发展和创新基金（NRDI基金）FK 138929；CF信托战略研究联盟基金NU-000600

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来源期刊

Journal of Cystic Fibrosis 医学-呼吸系统

CiteScore

10.10

自引率

13.50%

发文量

1361

审稿时长

50 days

期刊介绍： The Journal of Cystic Fibrosis is the official journal of the European Cystic Fibrosis Society. The journal is devoted to promoting the research and treatment of cystic fibrosis. To this end the journal publishes original scientific articles, editorials, case reports, short communications and other information relevant to cystic fibrosis. The journal also publishes news and articles concerning the activities and policies of the ECFS as well as those of other societies related the ECFS.