Growth hormone treatment for Prader-Willi syndrome: A review |
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| Affiliation: | 1. Facultad de Medicina, Unidad Docente Parc Taulí, Universitat Autònoma de Barcelona, Barcelona, España;2. Servicio de Endocrinología y Nutrición, Hospital Universitari Parc Tauli, , Sabadell, España;1. La Trobe University, Kingsbury Drive, Bundoora, Victoria 3086, Australia;2. The University of Melbourne, Grattan Street, Parkville, Victoria 3010, Australia;3. Eastern Health, 5 Arnold Street, Box Hill, Victoria 3128, Australia;1. Department of Restorative Dentistry, Faculty of Dentistry, Universidad de Concepción, Concepción, Chile;2. Friedrich-Alexander-Universität Erlangen-Nürnberg (FAU), Zahnklinik 1 – Zahnerhaltung und Parodontologie, Forschungslabor für dentale Biomaterialien, Erlangen, Germany;1. MIND Institute, University of California, Davis School of Medicine, Department of Psychiatry and Behavioral Sciences, Sacramento, CA, USA;2. MIND Institute, Genome Center, UC Davis School of Medicine, Department of Medical Microbiology and Immunology, Davis, CA, USA;3. Department of Psychiatry and Institute for Genomic Medicine, New York, NY, USA |
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| Abstract: | The Prader-Willi Syndrome (PWS) is a rare developmental disorder that contributed by multiple genes. Phenotypically, infants with PWS exhibit hypotonia and developmental delay, whilst older children and adults have cognitive impairments, neuropsychiatric symptoms, impaired motor development, neurological anomalies, endocrine dysfunctions like growth hormone (GH) deficiency, and hyperphagia that leads to obesity. Although mechanisms remain elusive, GH treatment has been recommended as the standard treatment for PWS children. In addition to better motor development, improved body composition and linear growth have been well established, but mental flexibility and behavioural problems remained largely untouched. This review will systemically analyze the recent clinical trials of GH treatment on PWS patients. The emphasis is on the mental and behavioural improvements by GH treatment, and a few concerns to initiate GH treatment. This review will finally propose possible future explorations on basic studies that may shed new light on clinical trials of GH treatment on PWS. |
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