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

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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.

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Acknowledgements

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.

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Authors and Affiliations

  1. Section of Medical Pathophysiology, Endocrinology, Department Experimental Medicine, Sapienza University of Rome, 00161, Rome, Italy

    C. Pascucci, L. Gnessi, C. Lubrano & A. Lenzi

  2. Cystic Fibrosis Unit, Bambino Gesù Children’s Hospital, Rome, Italy

    R. V. De Biase & D. Savi

  3. Department of Public Health and Infectious Diseases, Adult Cystic Fibrosis Center, Sapienza University of Rome, 00185, Rome, Italy

    D. Savi

  4. Pediatrics Department, Cystic Fibrosis Regional Center, Sapienza University of Rome, Rome, Italy

    S. Quattrucci

Authors
  1. C. Pascucci
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  2. R. V. De Biase
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  3. D. Savi
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  4. S. Quattrucci
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  5. L. Gnessi
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  6. C. Lubrano
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  7. A. Lenzi
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Contributions

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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Conflict of interest

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

Ethical approval

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

Informed consent

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). https://doi.org/10.1007/s40618-019-01051-4

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  • DOI: https://doi.org/10.1007/s40618-019-01051-4

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