Division of Pulmonary Medicine, Department of Pediatrics, Boston Children's Hospital, 300 Longwood Avenue, BCH 3121, Boston, MA 02115, USA; Harvard Medical School, Harvard University, 25 Shattuck Street, Boston, MA 02115, USA.
Division of Pulmonary Medicine, Department of Pediatrics, Boston Children's Hospital, 300 Longwood Avenue, BCH 3121, Boston, MA 02115, USA; Harvard Medical School, Harvard University, 25 Shattuck Street, Boston, MA 02115, USA.
Clin Chest Med. 2024 Sep;45(3):625-637. doi: 10.1016/j.ccm.2024.03.006. Epub 2024 Apr 30.
The landscape of care for children with cystic fibrosis (CF), a genetic disorder of chloride transport with multisystem manifestations including inspissated mucus, recurrent sinopulmonary infections, obstructive lung disease, and exocrine pancreatic insufficiency, is rapidly changing. Early diagnosis via newborn screening enabling timely nutritional support, chronic therapies to improve mucociliary clearance, and prompt treatment of pulmonary infections have improved overall outcomes in children with CF. More widespread availability of novel cystic fibrosis transmembrane conductance regulator modulator therapies for children continues to revolutionize pediatric CF care.However, significant challenges exist to optimize care and outcomes for all children with CF.
囊性纤维化（CF）是一种氯离子转运的遗传性疾病，其多系统表现包括黏液稠厚、反复的鼻窦和肺部感染、阻塞性肺病和外分泌胰腺功能不全。目前，该病患儿的治疗护理格局正在迅速改变。通过新生儿筛查做到早期诊断，从而及时给予营养支持，采用慢性治疗方法改善黏液纤毛清除功能，并及时治疗肺部感染，这些措施均改善了 CF 患儿的整体预后。新型囊性纤维化跨膜电导调节因子调节剂疗法在儿童中的广泛应用也不断推动儿科 CF 护理的发展。然而，要优化所有 CF 患儿的护理和结局，仍面临着重大挑战。
