The validated French CFAbd-Score reveals a lower burden of gastrointestinal symptoms in patients on Elexacaftor/Tezacaftor/Ivacaftor.

OBJECTIVES

Multiorgan abdominal involvement is a hallmark of Cystic fibrosis (CF). The CFAbd-Score© is the first CF-specific gastrointestinal patient reported outcome-measure (PROM) developed following FDA-guidelines. The PROM has proved to sensitively differentiate people with CF (pwCF) from healthy controls (HC). Furthermore, higher Scores in pwCF are associated with pancreatic insufficiency and history of surgery. Recently, in two large cohorts of pwCF, the PROM revealed significant and clinically meaningful improvements in abdominal symptoms (AS) in pwCF treated with Elexacaftor-Tezacaftor-Ivacaftor (ETI). In the present publication, we aim to validate the translated, linguistically and cross-culturally adapted French version of the CFAbd-Score©.

METHODS

Validation was performed following two forward translations and a review process by a German professional medical translator. The reviewed consensus version was psychometrically evaluated for validity and reliability in French-speaking pwCF and HC cohorts, as well as in pwCF with and without ETI.

RESULTS

A total of 64 French-speaking pwCF (mean age: 12.5 years) and 58 HC were included. Median CFAbd-Score© in pwCF without ETI was significantly higher than in HC (18.2 vs. 8.7 points/p < 0.01), while Scores from ETI-treated pwCF were almost as low as the HC-range in many domains. Scores from ETI-treated and nontreated pwCF differed significantly (8.4 vs. 18.2 points/p < 0.01).

CONCLUSION

The French version of the CFAbd-Score has proved to be reliable and sensitive to capture AS in French-speaking pwCF and to distinguish pwCF from HC. Furthermore, consistent with previous findings, the PROM sensitively detects ETI-related differences in AS of pwCF with and without ETI therapy, and thus can be used in research and clinical routine.