Advances in Care and Outcomes for Children with Cystic Fibrosis

Bob Z Sun; Gregory S Sawicki

doi:10.1016/j.ccm.2024.03.006

Advances in Care and Outcomes for Children with Cystic Fibrosis

Clin Chest Med. 2024 Sep;45(3):625-637. doi: 10.1016/j.ccm.2024.03.006. Epub 2024 Apr 30.

Authors

Bob Z Sun¹, Gregory S Sawicki²

Affiliations

¹ 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.
² 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. Electronic address: [email protected].

PMID: 39069326
DOI: 10.1016/j.ccm.2024.03.006

Abstract

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.

Keywords: CFTR modulator; CRMS; Cystic fibrosis; Newborn screening; Pediatrics.

Publication types

Review

MeSH terms

Child
Cystic Fibrosis Transmembrane Conductance Regulator / genetics
Cystic Fibrosis* / diagnosis
Cystic Fibrosis* / physiopathology
Cystic Fibrosis* / therapy
Humans
Infant, Newborn
Neonatal Screening

Substances

Cystic Fibrosis Transmembrane Conductance Regulator