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Sequelae of GH Treatment in Children with PWS
Maïthé Tauber1, Gwenaelle Diene2, Catherine Molinas1
1Centre de référence du syndrome de Prader-Willi, Hôpital des Enfants, CHU Toulouse, France, Axe Pédiatrique du CIC 9302/INSERM. Hôpital des Enfants, Toulouse, France, Centre de Physiopathologie de Toulouse-Purpan, Université de Toulouse, CNRS UMR 5282, INSERM UMR 1043, Université Paul Sabatier, Toulouse, France.
Abstract:
More than 15 years after rGH was granted marketing authorization for children with PWS, a review of the sequelae, side effects and safety issues of rGH therapy is timely. The publications on issues concerning respiratory function, glucose metabolism, fat mass, and scoliosis at baseline and with rGH treatment are herein presented. We discuss the impact of rGH side effects, make proposals to prevent or treat them, and emphasise the remaining questions and perspectives. As a whole, the benefit /risk ratio is positive, although questions are raised about the role of GH in premature pubarche and its long-term effects, particularly the potential long-term oncogenic risk. The organisation of care in dedicated or reference centres at the national and European level will facilitate the collection and analysis of data and serve as a paradigm for long-term follow-up.
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