Eight Years of Growth Hormone Treatment in Children With Prader-Willi Syndrome: Maintaining the Positive Effects

N. E. Bakker, R. J. Kuppens, E. P. C. Siemensma, R. F. A. Tummers-de Lind van Wijngaarden, D. A. M. Festen, G. C. B. Bindels-de Heus, G. Bocca, D. A. J. P. Haring, J. J. G. Hoorweg-Nijman, E. C. A. M. Houdijk, P. E. Jira, L. Lunshof, R. J. Odink, W. Oostdijk, J. Rotteveel, E. J. Schroor, A. A. E. M. van Alfen, M. van Leeuwen, E. van Pinxteren-Nagler, H. van WieringenR. C. F. M. Vreuls, N. Zwaveling-Soonawala, M. A. J. de Ridder, A. C. S. Hokken-Koelega

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Background: The most important reason for treating children with Prader-Willi syndrome (PWS) with GH is to optimize their body composition. Objectives: The aim of this ongoing study was to determine whether long-term GH treatment can counteract the clinical course of increasing obesity in PWS by maintaining the improved body composition brought during early treatment. Setting: This was a multicenter prospective cohort study. Methods: We have been following 60 prepubertal children for 8 years of continuous GH treatment (1 mg/m(2)/d congruent to 0.035 mg/kg/d) and used the same dual-energy x-ray absorptiometry machine for annual measurements of lean body mass and percent fat. Results: After a significant increase during the first year of GH treatment (P <.0001), lean body mass remained stable for 7 years at a level above baseline (P <.0001). After a significant decrease in the first year, percent fat SD score (SDS) and body mass index SDS remained stable at a level not significantly higher than at baseline (P = .06, P = .14, resp.). However, body mass index SDSPWS was significantly lower after 8 years of GH treatment than at baseline (P <.0001). After 8 years of treatment, height SDS and head circumference SDS had completely normalized. IGF-1 SDS increased to +2.36 SDS during the first year of treatment (P <.0001) and remained stable since then. GH treatment did not adversely affect glucose homeostasis, serum lipids, blood pressure, and bone maturation. Conclusion: This 8-year study demonstrates that GH treatment is a potent force for counteracting the clinical course of obesity in children with PWS
Original languageEnglish
Pages (from-to)4013-4022
JournalJournal of clinical endocrinology and metabolism
Issue number10
Publication statusPublished - 2013

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