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Endocrine Abstracts (2021) 73 AEP493 | DOI: 10.1530/endoabs.73.AEP493

1Erasmus University Medical Center, Internal Medicine, Division of Endocrinology, Rotterdam, Netherlands; 2Universidade de Sao Paulo, Pediatric Endocrinology Unit, Sao Paulo, Brazil; 3Hôpital Universitaire Necker Enfants Malades, Université Paris Descartes, Pediatric Endocrinology, Gynecology and Diabetology, Centre de Référence des Pathologies Gynécologiques Rares et des Maladies Endocriniennes Rares de la Croissance et du Développement, Paris, France; 4Universidade de Sao Paulo, Sao Paulo, Brazil


Background

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 improves physical health, cognition and quality of life (QoL). GH treatment is standard of care in PWS children, but in adults this is not the case. A systematic review and meta-analysis was conducted to provide a concise overview of the current knowledge on GH treatment in adults with PWS.

Methods

The databases Medline, Embase and Cochrane Central Register of Controlled Trials were sought for studies on the efficacy or effectiveness of GH treatment for adults with PWS. Randomized controlled trials (RCTs) and non-randomized (un)controlled trials (NRCTs) that reported data for adults with PWS who received GH treatment for at least six months were selected. Data on body composition, body mass index (BMI), cardiovascular endpoints, bone, cognitive function, QoL and safety were extracted.

Results

Nine RCTs and 19 NRCTs were included. Body composition improved during GH treatment with an increase in mean (95% CI) lean body mass of 1.72 kg (0.33–3.10 kg) for 258 person-years of follow-up, and a reduction of mean (95% CI) fat mass of –1.94% (–3.30% to –0.57%) for 223 person-years of follow-up. BMI, low-density lipoprotein levels and bone mineral density did not change during GH treatment. GH treatment was safe for both GH naïve adults with PWS and for adults previously treated with GH.

Conclusion

GH treatment is safe and improves body composition in adults with PWS. As poor body composition plays a key role in the high cardiovascular morbidity of adults with PWS, these data suggest that GH treatment might reduce long-term cardiovascular complications in this vulnerable patient group.

Volume 73

European Congress of Endocrinology 2021

Online
22 May 2021 - 26 May 2021

European Society of Endocrinology 

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