C Pascucci1, R V De Biase2, D Savi2,3, S Quattrucci4, L Gnessi5, C Lubrano5, A Lenzi5. 1. Section of Medical Pathophysiology, Endocrinology, Department Experimental Medicine, Sapienza University of Rome, 00161, Rome, Italy. chiarapascucci@libero.it. 2. Cystic Fibrosis Unit, Bambino Gesù Children's Hospital, Rome, Italy. 3. Department of Public Health and Infectious Diseases, Adult Cystic Fibrosis Center, Sapienza University of Rome, 00185, Rome, Italy. 4. Pediatrics Department, Cystic Fibrosis Regional Center, Sapienza University of Rome, Rome, Italy. 5. Section of Medical Pathophysiology, Endocrinology, Department Experimental Medicine, Sapienza University of Rome, 00161, Rome, Italy.
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.
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 CFpatients 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; A M Isidori; C Lubrano; L Gnessi; A Lenzi Journal: J Endocrinol Invest Date: 2017-11-02 Impact factor: 4.256
Authors: M Poggi; C Pascucci; S Monti; P Pugliese; C Lauri; G Amodeo; G Girelli; V Toscano Journal: J Endocrinol Invest Date: 2010-02-15 Impact factor: 4.256
Authors: Claire E Wainwright; J Stuart Elborn; Bonnie W Ramsey; Gautham Marigowda; Xiaohong Huang; Marco Cipolli; Carla Colombo; Jane C Davies; Kris De Boeck; Patrick A Flume; Michael W Konstan; Susanna A McColley; Karen McCoy; Edward F McKone; Anne Munck; Felix Ratjen; Steven M Rowe; David Waltz; Michael P Boyle Journal: N Engl J Med Date: 2015-05-17 Impact factor: 91.245
Authors: Valentine Sergeev; Frank Y Chou; Grace Y Lam; Christopher Michael Hamilton; Pearce G Wilcox; Bradley S Quon Journal: Ann Am Thorac Soc Date: 2020-02