Recent advances in the early treatment of cystic fibrosis: Bridging the gap to highly effective modulator therapy

Abstract

Highly effective modulator therapy (HEMT) for cystic fibrosis (CF) has been touted as one of the greatest advances to date in CF care. As these therapies are now available for many older children and adults with CF, marked improvement of their nutritional status, pulmonary and gastrointestinal symptoms has been observed. However, most infants and younger children are not current candidates for HEMT due to age and/or cystic fibrosis transmembrane conductance regulator (CFTR) mutation. For these young children, it is essential to provide rigorous monitoring and care to avoid potential disease sequelae while awaiting HEMT availability. The following article highlights recent advances in the care of infants and young children with CF with regard to surveillance and treatment of nutritional, pulmonary, and gastrointestinal disorders. Recent clinical trials in this population are also reviewed.

Lung structural and functional impairments in young children with cystic fibrosis diagnosed following newborn screening – A nationwide observational study

“…This proportion is lower than in previous studies, which have shown that between 50 and 70 % of young children with CF have abnormal LCI values [31]. One influencing factor might be the advances in supportive therapies in the past years [32]. Our MBW data were also analyzed using the most recently updated software version for the Eco Medics device, which includes a correction for a previously unknown sensor error resulting in over-estimated LCI values [33].…”