Impact of CFTR-modulating drugs on GH-IGF-1 axis impairment in adult patients with cystic fibrosis.

Abstract

INTRODUCTION:

A new class of drugs in the treatment of cystic fibrosis (CF) includes two agents: lumacaftor, which corrects CFTR channel protein, and ivacaftor, which increases CFTR channel activity. In our previous study we recruited 50 stable adults with CF and 16 of them showed growth hormone deficit (GHD): 7 patients severe and 9 patients partial GHD.

MATERIAL AND METHODS:

We decided to re-evaluate ten patients with the GHRH + arginine test of whom only five were treated with lumacaftor/ivacaftor.

RESULTS:

All CF patients in therapy with lumacaftor/ivacaftor showed a marked improvement in GHD. Two patients moved from a severe GHD to a normal response to the GH/IGF-1 axis test, and three patients who had partial GHD moved to normal response.

CONCLUSION:

The pituitary gland may be damaged by CF disease and could benefit of the action of correcting drugs.

• Authors: C Pascucci, R V De Biase, D Savi, S Quattrucci, L Gnessi, C Lubrano, A Lenzi
• Journal: Journal of endocrinological investigation (J Endocrinol Invest) Vol. 42 Issue 11 Pg. 1361-1363 (Nov 2019) ISSN: 1720-8386 [Electronic] Italy
• PMID: 31006073 (Publication Type: Journal Article)

Chemical References

• Aminophenols
• Aminopyridines
• Benzodioxoles
• CFTR protein, human
• Drug Combinations
• IGF1 protein, human
• Quinolones
• lumacaftor, ivacaftor drug combination
• Human Growth Hormone
• Cystic Fibrosis Transmembrane Conductance Regulator
• Insulin-Like Growth Factor I

Topics

• Adult
• Aminophenols (therapeutic use)
• Aminopyridines (therapeutic use)
• Benzodioxoles (therapeutic use)
• Cystic Fibrosis (drug therapy, genetics, pathology)
• Cystic Fibrosis Transmembrane Conductance Regulator (genetics)
• Drug Combinations
• Female
• Follow-Up Studies
• Growth Disorders (pathology, prevention & control)
• Human Growth Hormone (metabolism)
• Humans
• Insulin-Like Growth Factor I (metabolism)
• Male
• Middle Aged
• Mutation
• Prognosis
• Quinolones (therapeutic use)