Beneficial Effects of Growth Hormone Treatment on Cognition in Children with Prader-Willi Syndrome: A Randomized Controlled Trial and Longitudinal Study

Elbrich P. C. Siemensma*, Roderick F. A. Tummers-de Lind van Wijngaarden, Dederieke A. M. Festen, Zyrhea C. E. Troeman, A. A. E. M. (Janielle) van Alfen-van der Velden, Barto J. Otten, Joost Rotteveel, Roelof J. H. Odink, G. C. B. (Karen) Bindels-de Heus, Mariette van Leeuwen, Danny A. J. P. Haring, Wilma Oostdijk, Gianni Bocca, E. C. A. Mieke Houdijk, A. S. Paul van Trotsenburg, J. J. Gera Hoorweg-Nijman, Hester van Wieringen, Rene C. F. M. Vreuls, Petr E. Jira, Eelco J. SchroorEvelyn van Pinxteren-Nagler, Jan Willem Pilon, L. (Bert) Lunshof, Anita C. S. Hokken-Koelega

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

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Background: Knowledge about the effects of GH treatment on cognitive functioning in children with Prader-Willi syndrome (PWS) is limited.

Methods: Fifty prepubertal children aged 3.5 to 14 yr were studied in a randomized controlled GH trial during 2 yr, followed by a longitudinal study during 4 yr of GH treatment. Cognitive functioning was measured biennially by short forms of the WPPSI-R or WISC-R, depending on age. Total IQ (TIQ) score was estimated based on two subtest scores.

Results: During the randomized controlled trial, mean SD scores of all subtests and mean TIQ score remained similar compared to baseline in GH-treated children with PWS, whereas in untreated controls mean subtest SD scores and mean TIQ score decreased and became lower compared to baseline. This decline was significant for the Similarities (P = 0.04) and Vocabulary (P = 0.03) subtests. After 4 yr of GH treatment, mean SD scores on the Similarities and Block design subtests were significantly higher than at baseline (P = 0.01 and P = 0.03, respectively), and scores on Vocabulary and TIQ remained similar compared to baseline. At baseline, children with a maternal uniparental disomy had a significantly lower score on the Block design subtest (P = 0.01) but a larger increment on this subtest during 4 yr of GH treatment than children with a deletion. Lower baseline scores correlated significantly with higher increases in Similarities (P = 0.04) and Block design (P <0.0001) SD scores.

Conclusions: Our study shows that GH treatment prevents deterioration of certain cognitive skills in children with PWS on the short term and significantly improves abstract reasoning and visuospatial skills during 4 yr of GH treatment. Furthermore, children with a greater deficit had more benefit from GH treatment. (J Clin Endocrinol Metab 97: 2307-2314, 2012)

Original languageEnglish
Pages (from-to)2307-2314
Number of pages8
JournalJournal of Clinical Endocrinology and Metabolism
Issue number7
Publication statusPublished - Jul-2012


  • GH

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