Validation of the CFAbd-Score.kid, a Novel gastrointestinal patient reported outcome measure, specific for children with cystic fibrosis.
作者信息
Sadrieh Pauline, Bechinger Lilith, Duckstein Franziska, Barucha Anton, Nährlich Lutz, Eickmeier Olaf, van Dullemen Suzanne, Kemp Daniel, Mühl Berit, Eschenhagen Patience, Schwarz Carsten, Graepler-Mainka Ute, Zagoya Carlos, Mainz Jochen G
机构信息
Cystic Fibrosis-Center, Klinikum Westbrandenburg, Campus Brandenburg an der Havel, Germany; Medizinische Hochschule Brandenburg (MHB), Universitätsklinikum, Brandenburg an der Havel, Germany.
Cystic Fibrosis-Center, Klinikum Westbrandenburg, Campus Brandenburg an der Havel, Germany; Medizinische Hochschule Brandenburg (MHB), Universitätsklinikum, Brandenburg an der Havel, Germany; Department of Gastroenterology, Brandenburg Medical School (Theodor Fontane), Brandenburg an der Havel, Germany.
出版信息
J Cyst Fibros. 2025 Sep;24(5):886-893. doi: 10.1016/j.jcf.2025.07.010. Epub 2025 Jul 23.
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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