Improved Mental and Motor Development During 3 Years of GH Treatment in Very Young Children With Prader-Willi Syndrome.

CONTEXT

Infants and toddlers with Prader-Willi syndrome (PWS) have mental and motor developmental delay. Short-term data suggest a positive effect of GH on mental and motor development in infants and children with PWS. There are, however, no longer-term results about the effects of GH treatment on mental and motor development.

OBJECTIVE

To investigate the longer-term effects of GH on psychomotor development in infants and toddlers with PWS and the effect of age at start of GH treatment on psychomotor development.

DESIGN

Prospective cohort study during 3 years of GH treatment.

SETTING

The PWS Reference Center in the Netherlands.

INTERVENTION

All children were treated with GH 1 mg/m2/d (≈0.035 mg/kg/d).

MAIN OUTCOME MEASURES

Mental and motor developmental age assessed with Bayleys Scales of Infant Development II and expressed as percentage of the expected development (100%).

RESULTS

During 3 years of GH, mean (SEM) mental development increased from 58.1% (2.8) at baseline to 79.6% (3.7), and motor development increased from 41.9% (2.9) to 78.2% (3.9; both P < 0.01). A lower baseline psychomotor development and a younger age at start of GH treatment were associated with a higher increase in mental and motor development (P < 0.01).

CONCLUSIONS

Mental and motor development increased significantly during 3 years of GH treatment, reducing the gap between infants with PWS and healthy peers. A younger age at start of GH treatment leads to greater improvement in psychomotor development.