Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2025
Validation of the CFAbd-Score.kidⒸ, a Novel gastrointestinal patient reported outcome measure, specific for children with cystic fibrosis.
СтатьяВалидационное исследованиеНазвание и аннотация: Перевод пока не подготовлен
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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
Аннотация
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.
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.