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



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.


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.


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

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The authors would like to express their gratitude to Gaius Stern, Ph.D., U.C. Berkeley. For English translation support we had on this manuscript.

Author information




CP and RVDB conceived study. CP recruited patients and conducted the trial procedures. CP, SQ and RVDB analyzed and interpreted the data. CP, DS, SQ and RVDB drafted the manuscript. LG, CL and AL revised the manuscript. All authors read and approved the final manuscript.

Corresponding author

Correspondence to C. Pascucci.

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The authors declare that they have no conflicts of interest in this work.

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The present study was approved by the ethics committee of the University and adhered to the tenets of the Declaration of Helsinki.

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Additionally, the written informed consents were signed by all participants.

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Pascucci, C., De Biase, R.V., Savi, D. et al. Impact of CFTR-modulating drugs on GH-IGF-1 axis impairment in adult patients with cystic fibrosis. J Endocrinol Invest 42, 1361–1363 (2019).

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  • Growth hormone
  • Insulin-like growth factor-1
  • Cystic fibrosis
  • Lumacaftor
  • Ivacaftor
  • Adult patients