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Growth failure and treatment in cystic fibrosis

Trang N Le1, Abeer Anabtawi2, Melissa S Putman3

  • 1Departments of Internal Medicine and Pediatrics, Division of Endocrinology, Virginia Commonwealth University, Richmond, VA, USA.

Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society
|November 5, 2019
PubMed

Insights

Cystic fibrosis (CF) impacts growth, with height lagging behind weight gains despite improved care. New insights into CFTR

Area of Science:

  • Pediatric Endocrinology
  • Pulmonology
  • Genetics

Background:

  • Poor growth is a hallmark of cystic fibrosis (CF), impacting lung function and overall health.
  • While nutrition and pulmonary care have improved weight gain in CF patients, height deficits persist, especially in those with severe CFTR mutations.
  • The chronic inflammatory state in CF affects the growth hormone-IGF-1 axis and bone development.

Purpose of the Study:

  • To review the factors influencing growth in cystic fibrosis patients.
  • To explore the role of CFTR in bone health and the potential of CFTR modulators for growth.
  • To discuss current and emerging treatments for growth deficits in CF.

Main Methods:

  • Literature review of studies on growth in cystic fibrosis.
  • Analysis of factors affecting the growth hormone-IGF-1 axis and bone growth in CF.
  • Examination of the impact of CFTR mutations and modulators on growth outcomes.

Main Results:

  • Height gains have not kept pace with weight improvements in children with CF.
  • Severe CFTR mutations are associated with more significant growth impairments.
  • Emerging data suggest direct effects of CFTR on bone, with implications for CFTR modulator therapies.

Conclusions:

  • Optimizing growth in CF requires addressing multifactorial influences, including inflammation and CFTR function.
  • CFTR modulators may offer new avenues for improving growth in cystic fibrosis.
  • Further research is needed to fully understand and treat growth deficits in CF.

Keywords:
Cystic fibrosisGrowthGrowth delayShort stature

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