Prevention of cystic fibrosis (CF) lung disease by the early use of triple combination elexacaftor/tezacaftor/ivacaftor (ETI) will be key in the future standard of care of young children with CF [1, 2]. Almost every paediatrician agrees with this assumption, and evidence has been provided by clinical trials that it slows disease progression in school-aged children with CF [3, 4] and has a great potential to limit bronchiectasis development [2]. As patients enrolled in clinical trials are often highly selected, translation of data obtained from these clinical trials to the "real world" can be limited, to the extent that children commencing highly effective modulator therapy may differ somewhat from the trial participants in whom the benefits of ETI were proven.

通过早期使用 elexacaftor/tezacaftor/ivacaftor (ETI) 三联组合来预防囊性纤维化 (CF) 肺部疾病将是未来 CF 幼儿标准护理的关键 [1, 2]。几乎每位儿科医生都同意这一假设，并且临床试验提供的证据表明，它可以减缓学龄 CF 儿童的疾病进展 [3, 4]，并且具有限制支气管扩张发展的巨大潜力 [2]。由于参加临床试验的患者往往经过严格挑选，从这些临床试验中获得的数据到“现实世界”的转化可能受到限制，以至于开始高效调节剂治疗的儿童可能与受益的试验参与者有所不同。 ETI 已被证明。