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

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.

Deregulation of the growth hormone/insulin-like growth factor-1 axis in adults with cystic fibrosis.

PURPOSE: Patients with cystic fibrosis (CF) present with signs and symptoms that overlap with those of adult growth hormone deficiency (GHD) syndrome: loss of muscle mass, bone fragility and lower stress tolerance. In literature, the prevalence of GHD in pediatric CF patients is higher than general population, but these studies have been performed on children with growth delay. To our knowledge, there are no studies on adult patients. The aim of this paper is to evaluate GH-IGF1 axis in an adult CF population. METHODS: Fifty clinically stable adult patients, 30 males; age 36 ± 2 years; BMI 21.39 ± 0.22 kg/m2 and FEV1 67 ± 4% were studied. Data regarding glycometabolic status and results of pituitary, thyroid, parathyroid, gonadal and adrenal function tests were recorded. All patients underwent a GH releasing hormone (GHRH) + Arginine stimulation test to confirm a GHD. RESULTS: GHRH + Arginine test revealed the presence of GHD in 16 patients (32%); specifically 7 patients had a severe deficiency and 9 a partial deficiency. CONCLUSIONS: Adult patients with CF may show GHD. These patients should be followed over time to assess if the GHD could impact the clinical progression of CF.

Clinical and microbiological monitoring of Cystic Fibrosis patients, three years of follow-up via Tele-Medicine: an empirical research.

OBJECTIVES: Evaluation of the effectiveness of home care through a telemonitoring system in reducing the incidence of new colonization by Pseudomonas Aeruginosa in a population of patients with Cystic Fibrosis (CF) followed by the CF clinic of the Bambino Gesù Hospital in Rome over a period of 36 months. MATERIALS AND METHODS: Two groups of patients were recruited, homogeneous for age, sex, BMI, FEV1, prevalence of CF-related Diabetes and CF-related Hepatopathy, access to new therapies with modulators: a) an IN group (N = 44 ) followed through a home telemonitoring system, b) an OUT control group (N = 110) followed according to the standards of care. The following parameters were detected for all patients: pulmonary colonization of the lungs, number and type of hospital admissions, respiratory function, BMI. RESULT: The OUT group had a statistically significant increase in the prevalence of Pseudomonas Aeruginosa infections during the observation period. Furthermore, a significant decrease in lung function assessed through FEV1 was also observed in the OUT group. CONCLUSION: Adolescent and adult patients belonging to the CF center who are not followed through the dedicated home telemonitoring service show, in the three-year period 2017-19, an increase in Pseudomonas Aeruginosa infections and a greater decrease in respiratory function. The use of telemedicine in CF is therefore an effective system not only in monitoring the disease but also as a treatment strategy, in the context of an evolving multidisciplinary model. As advantages, telemedicine can reduce the number of Pseudomonas Aeruginosa lung infections and the greater stability of respiratory function over time.

Clinical effects of diet supplementation with DHA in pediatric patients suffering from cystic fibrosis.

AIM: Cystic fibrosis (CF) patients present an altered fatty acid (FA) metabolism characterized by imbalance in the arachidonic/docosohexasenoic acid (AA/DHA) ratio in favour of the former which can contribute to the increase in pulmonary inflammation. The present study aims to assess respiratory, nutritional, clinical and laboratory parameters, and inflammatory markers after six months of DHA supplementation in paediatric patients suffering from CF. METHODS: A dose of 1 g/10 kg/die was administered to ten CF patients of paediatric age for the first month and 250 mg/10 kg/die for the remaining 5 months. We carried out follow-ups at T0 (baseline), T6 (after six months of the diet) and T12 (six months after supplementation was interrupted) during which respiratory, nutritional, clinical and laboratory parameters were assessed. RESULTS: After six months of DHA supplementation inflammatory marker levels had diminished: interleukin 8 (IL-8) and Tumour Necrosis Factor Alfa (TNF-α) in serum, and calprotectin in stools. In addition, auxometric parameters were improved as was the clinical condition of patients, who tolerated DHA well. CONCLUSION: Dietetic integration with DHA seems to improve clinical condition and the inflammatory pulmonary and intestinal state of pediatric patients suffering from CF.