Behavior in children with Prader–Willi syndrome before and during growth hormone treatment: a randomized controlled trial and 8-year longitudinal study

Abstract The aim of this study was to investigate the effect of growth hormone treatment on adaptive functioning in children with Prader-Willi syndrome. Vineland Adaptive Behavior Scale (VABS) was assessed during a randomized controlled trial (RCT) and after 7 years of growth hormone treatment. In the RCT, 75 children (42 infants and 33 prepubertal children) with Prader-Willi syndrome were included. Subsequently, 53 children were treated with long-term growth hormone. Our study demonstrates a marked delay in adaptive functioning in infants and children with Prader-Willi syndrome, which was associated with older age and lower intelligence. Results of the repeated measurements show that the earlier growth hormone treatment was started during infancy, the better the adaptive skills were on the long-term.

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

The Journal of Clinical Endocrinology & Metabolism ◽

10.1210/jc.2012-1182 ◽

2012 ◽

Vol 97 (7) ◽

pp. 2307-2314 ◽

Cited By ~ 85

Author(s):

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 ◽

...

Keyword(s):

Randomized Controlled Trial ◽

Longitudinal Study ◽

Cognitive Functioning ◽

Cognitive Skills ◽

Controlled Trial ◽

Block Design ◽

Uniparental Disomy ◽

Prader Willi Syndrome ◽

Gh Treatment ◽

Randomized Controlled

Abstract 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.

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