Validation of the CFAbd-Score.kid©, a Novel gastrointestinal patient reported outcome measure, specific for children with cystic fibrosis.

IF 6 2区医学 Q1 RESPIRATORY SYSTEM

Journal of Cystic Fibrosis Pub Date : 2025-07-23 DOI:10.1016/j.jcf.2025.07.010

Pauline Sadrieh, Lilith Bechinger, Franziska Duckstein, Anton Barucha, Lutz Nährlich, Olaf Eickmeier, Suzanne van Dullemen, Daniel Kemp, Berit Mühl, Patience Eschenhagen, Carsten Schwarz, Ute Graepler-Mainka, Carlos Zagoya, Jochen G Mainz

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引用次数: 0

Abstract

Background: Abdominal symptoms (AS) relevantly impair quality of life (QoL) in people with Cystic fibrosis (CF). Following FDA guidelines, we previously developed and validated the CFAbd-Score©, the first CF-specific gastrointestinal patient-reported outcome-measure (PROM) for people with CF. Therewith, we demonstrated that AS significantly and markedly decrease during treatment with the new game-changing CF-therapy elexacaftor-tezacaftor-ivacaftor (ETI). However, for children with CF (cwCF), a pediatric-focused PROM could improve self- or proxy-assisted reporting of AS.

Aims: Development and validation of the CFAbd-Score.kid©, the pediatric version of the CFAbd-Score©, following FDA- and COSMIN-guidelines, anticipating ETI approval in younger cwCF.

Methods: We iteratively developed the CFAbd-Score.kid© together with CF-specialists from different fields, cwCF and their proxies. We implemented pictograms and easy language to optimize comprehension of questions by cwCF aged <12years, as well as adapted response strategies. We devised a scoring algorithm, weighting items (n = 29) and domains (n = 5) differently, to optimize the PROM´s sensitivity (range: 0-100pts).

Results: In 5 German CF-centers, n = 102 cwCF (7.6 ± 2.3yrs) completed n = 257 CFAbd-Score.kid© questionnaires. For known-groups validity analysis, n = 72 healthy children (HC) were included. Good to excellent test-retest reliability was observed (intraclass correlation-coefficient=0.89, p < 0.001). Median total CFAbd-Score.kid© resulted significantly higher in cwCF, compared to HC (16.2 vs.10.1pts, p < 0.01). CwCF also scored significantly higher in domains "Disorders of bowel movement", "Disorders of appetite" and "Quality of life".

Conclusion: The novel CFAbd-Score.kid©, well accepted among children, allows recording of AS in cwCF. The total CFAbd-Score.kid© and three of its five domains reveal a significantly and markedly elevated burden of AS in cwCF, compared to HC.

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cfab - score的验证。kid©，一种新型胃肠道患者报告的结果测量，专门针对患有囊性纤维化的儿童。

背景：腹腔症状（AS）与囊性纤维化（CF）患者的生活质量（QoL）相关。根据FDA的指导方针，我们之前开发并验证了CFAbd-Score©，这是CF患者的第一个CF特异性胃肠道患者报告的结果测量（PROM）。因此，我们证明了在使用新的改变游戏规则的CF治疗elexaftor - tezactor -ivacaftor （ETI）治疗期间，AS显着显著减少。然而，对于CF患儿（cwCF），以儿科为中心的PROM可以改善自我或代理辅助的AS报告。目的：开发和验证cfabd评分。child©，CFAbd-Score©的儿科版本，遵循FDA和cosmin指南，预计在较年轻的cwCF中获得ETI批准。方法：对cfabd评分进行迭代开发。©与来自不同领域的cf专家，cwCF及其代理一起。结果：在5个德国cf中心中，n = 102名cwCF（7.6±2.3年）完成n = 257名CFAbd-Score。孩子©问卷。已知组效度分析纳入健康儿童（HC） 72例。观察到良好至极好的重测信度（类内相关系数=0.89,p < 0.001）。总cfabd评分中位数。儿童©的cwCF显著高于HC （16.2 vs.10.1, p < 0.01）。CwCF在“排便障碍”、“食欲障碍”和“生活质量”方面的得分也明显更高。结论：CFAbd-Score。kid©，在儿童中广为接受，允许在cwCF中记录AS。总CFAbd-Score。child©及其5个结构域中的3个显示，与HC相比，cwCF患者的AS负担显著升高。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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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.