Following ETI therapy initiation, the German cohort ( n = 68) reached a highly significant 0.4-fold decline ( p < 0.001) in total CFAbd-Scores, in contrast to the UK cohort ( n = 39), where a 0.9-fold declined did not reach significance.
Comparison of changes in domains during therapy with ETI in pwCF to averages of CFAbd-Scores obtained from healthy controls showed a trend towards normalization during HEMT in calculated total scores, regarding “pain”, “disorders of bowel movement” and “GI-related QoL”, without fully reaching levels of HC during early phases of ETI therapy.