Anna G W Rosenberg1,2, Caroline De Gouveia Buff Passone3,4, Karlijn Pellikaan1,2, Durval Damiani3, Aart J Van Der Lely1, Michel Polak4,5, Wanderley Marques Bernardo6, Laura C G De Graaff1,2,5,7. 1. Internal Medicine, division of Endocrinology, Erasmus MC, University Medical Centre Rotterdam, The Netherlands. 2. Dutch Centre of Reference for Prader-Willi syndrome. 3. Pediatric Endocrinology Unit, Universidade de Sao Paulo, Sao Paulo, Brazil. 4. Pediatric Endocrinology, Gynecology and Diabetology, Centre de Référence des Pathologies Gynécologiques Rares et des Maladies Endocriniennes Rares de la Croissance et du Développement, Hôpital Universitaire Necker Enfants Malades, Université de Paris, Paris, France. 5. ENDO-ERN (European Reference Network on Rare Endocrine Conditions). 6. Universidade de Sao Paulo, Sao Paulo, Brazil. 7. Academic Centre for Growth, Erasmus MC, University Medical Centre Rotterdam, The Netherlands.
Abstract
CONTEXT: Features of Prader-Willi syndrome (PWS) overlap with features of growth hormone (GH) deficiency, like small hands and feet, short stature, increased body fat and low muscle mass and strength. In children with PWS, GH treatment (GHt) improves physical health and cognition. GHt has become standard of care in PWS children, but in adults this is not yet the case. OBJECTIVE: To provide an overview of the current knowledge on GHt in PWS adults. DATA SOURCE: Medline, Embase and Cochrane Central Register of Controlled Trials databases. STUDY SELECTION: Randomized controlled trials (RCTs) and non-randomized (un)controlled trials (NRCTs) that reported data for adults with PWS, who received GHt for at least six months. DATA EXTRACTION: Data on body composition, body mass index (BMI), cardiovascular endpoints, bone, cognitive function, quality of life and safety were extracted. DATA SYNTHESIS: Nine RCTs and 20 NRCTs were included. Body composition improved during 12 months of GHt with an increase in mean (95% CI) lean body mass of 1.95 kg (0.04 - 3.87 kg), and a reduction of mean (95% CI) fat mass of -2.23% (-4.10% to -0.36%). BMI, low-density lipoprotein cholesterol levels, fasting glucose levels and bone mineral density did not change during GHt. There were no major safety issues. CONCLUSION: GHt appears to be safe and improves body composition in adults with PWS. As poor body composition is closely linked to the observed high incidence of cardiovascular morbidity in adults with PWS, improving body composition might reduce cardiovascular complications in this vulnerable patient group.
CONTEXT: Features of Prader-Willi syndrome (PWS) overlap with features of growth hormone (GH) deficiency, like small hands and feet, short stature, increased body fat and low muscle mass and strength. In children with PWS, GH treatment (GHt) improves physical health and cognition. GHt has become standard of care in PWSchildren, but in adults this is not yet the case. OBJECTIVE: To provide an overview of the current knowledge on GHt in PWS adults. DATA SOURCE: Medline, Embase and Cochrane Central Register of Controlled Trials databases. STUDY SELECTION: Randomized controlled trials (RCTs) and non-randomized (un)controlled trials (NRCTs) that reported data for adults with PWS, who received GHt for at least six months. DATA EXTRACTION: Data on body composition, body mass index (BMI), cardiovascular endpoints, bone, cognitive function, quality of life and safety were extracted. DATA SYNTHESIS: Nine RCTs and 20 NRCTs were included. Body composition improved during 12 months of GHt with an increase in mean (95% CI) lean body mass of 1.95 kg (0.04 - 3.87 kg), and a reduction of mean (95% CI) fat mass of -2.23% (-4.10% to -0.36%). BMI, low-density lipoprotein cholesterol levels, fasting glucose levels and bone mineral density did not change during GHt. There were no major safety issues. CONCLUSION:GHt appears to be safe and improves body composition in adults with PWS. As poor body composition is closely linked to the observed high incidence of cardiovascular morbidity in adults with PWS, improving body composition might reduce cardiovascular complications in this vulnerable patient group.