Department of Paediatric Respiratory Medicine, Royal Brompton and Harefield NHS Foundation Trust, London, UK.
National Heart and Lung Institute, Imperial College London, London, UK; Department of Paediatric Respiratory Medicine, Royal Brompton and Harefield NHS Foundation Trust, London, UK.
Paediatr Respir Rev. 2020 Sep;35:99-102. doi: 10.1016/j.prrv.2020.04.003. Epub 2020 Apr 14.
Prior to the use of cystic fibrosis (CF) modulator therapy, exocrine pancreatic insufficiency in CF was thought to be irreversible. Improvement in pancreatic function on ivacaftor has been reported in open label studies in 1-5 year olds. The mechanism by which ivacaftor might improve exocrine pancreatic function is unclear. Although the effect of ivacaftor on pancreatic function may be more significant in younger children, evidence is mounting that there may still be potential for improvement in older children on long term therapy.
在使用囊性纤维化 (CF) 调节剂治疗之前，人们认为 CF 外分泌胰腺功能不全是不可逆转的。在 1-5 岁的开放性标签研究中，已经报道了 ivacaftor 可改善胰腺功能。然而，ivacaftor 改善外分泌胰腺功能的机制尚不清楚。尽管 ivacaftor 对胰腺功能的影响在年幼儿童中可能更为显著，但越来越多的证据表明，长期治疗的年长儿童仍可能有改善的潜力。
