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Pediatr Endocrinol Rev. 2018 Sep;16(Suppl 1):91-99. doi: 10.17458/per.vol16.2018.tdm.ghpraderwilli.

Growth Hormone Treatment for Prader-Willi Syndrome.

Author information

1
Centre de référence du syndrome de Prader-Willi, Hôpital des Enfants, CHU Toulouse, France, 2Axe Pédiatrique du CIC 9302/INSERM. Hôpital des Enfants, Toulouse, France, 3INSERM U1043, Centre de Physiopathologie de Toulouse Purpan, UPS, France, E-mail: tauber.mt@chu-toulouse.fr.
2
Centre de référence du syndrome de Prader-Willi, Hôpital des Enfants, CHU Toulouse, France, INSERM UMR 1027-Université Toulouse III Hôpital Paule de Viguier, Toulouse, France.
3
Centre de référence du syndrome de Prader-Willi, Hôpital des Enfants, CHU Toulouse, France, Axe Pédiatrique du CIC 9302/INSERM. Hôpital des Enfants, Toulouse, France, INSERM U1043, Centre de Physiopathologie de Toulouse Purpan, UPS, France.

Abstract

The European Marketing Authorization for recombinant human growth hormone (rhGH) in children with Prader-Willi syndrome was the first indication for metabolic and body composition effects in children. In the US it is indicated for short stature associated with PWS. Recombinant hGH is the first treatment for the PWS population and radically changed the care of these children by facilitating access to physicians who prescribe rhGH, mainly paediatric endocrinologists, and manage the organization of multidisciplinary care. Recombinant hGH treatment improved linear growth, body composition, and socialization not only in children but also in young adults. The pathophysiology of combined hormonal deficiencies including GH is starting to be unravelled. We now have to focus on co-morbidities that are not modified by rhGH treatment, such as feeding disorders and behaviour problems, to truly change the life of patients. The transition of care from adolescents to young adults also remains a challenge.

KEYWORDS:

Growth Hormone; Prader-Willi Syndrome

[Indexed for MEDLINE]

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