J Cyst Fibros. 2025 Sep 12:S1569-1993(25)01582-6. doi: 10.1016/j.jcf.2025.09.003. Online ahead of print.
ABSTRACT
BACKGROUND: Recently, elexacaftor/tezacaftor/ivacaftor (ETI), the turning point in the course of Cystic fibrosis (CF), was also approved for children with CF (cwCF) aged 6-11 years carrying at least one F508del mutation. Modulating the causative deficiency in the CF transmembrane conductance regulator channel was found to substantially improve the crucially affected respiratory and digestive CF-manifestations. In this regard, using the CFAbd-ScoreⒸ, we previously found that ETI decreases significantly abdominal symptoms (AS) in people with CF aged ≥ 12 years.
AIMS: Assessing changes in AS after ETI initiation in cwCF aged 6-11 years with the novel pediatric Patient-Reported Outcome-Measure CFAbd-Score.kidⒸ.
METHODS: The CFAbd-Score.kidⒸ, specially developed and validated for cwCF aged <12 years, implements pictograms, easy language and children oriented response strategies, comprising 29 CF-specific gastrointestinal items from five domains. Its scoring algorithm developed following FDA guidelines weights items and domains differently, reaching a maximum of 100 points. CwCF completing at least one questionnaire before ETI initiation and another one during ETI therapy were included.
RESULTS: In four German CF centers, a total of n = 52 cwCF (mean age 8.3 ± 2.2 years) were included, completing a total of n = 293 questionnaires. During ETI therapy, significant decreases were observed for mean total CFAbd-Score.kidⒸ (-31 %; p < 0.0001) as well as for mean sub-scores of “pain” (-26 %; p < 0.01), “QoL impairment” (-48 %; p < 0.01), “disorders of bowel movement” (-32 %; p ≤ 0.0001) and “disorders of appetite” (-42 %; p < 0.05).
CONCLUSION: Among cwCF aged 6-11 years, AS captured with the novel CFAbd-Score.kidⒸ significantly decreased during ETI treatment. Simultaneously, this proves that the recently validated CFAbd-Score.kidⒸ is sensitive to ETI induced changes in AS. The new questionnaire is being now implemented in clinical routine and international studies.
PMID:40946087 | DOI:10.1016/j.jcf.2025.09.003
