Mental and motor development before and during growth hormone treatment in infants and toddlers with Prader-Willi syndrome

• Published in: Clinical endocrinology (Oxford) Vol. 68; no. 6; pp. 919 - 925
• Main Authors: Festen, D. A. M., Wevers, M., Lindgren, A. C., Böhm, B., Otten, B. J., Wit, J. M., Duivenvoorden, H. J., Hokken-Koelega, A. C. S.
• Format: Journal Article
• Language: English
• Published: Oxford, UK Blackwell Publishing Ltd 01.06.2008; Blackwell
• Subjects: Biological and medical sciences; Body Composition - drug effects; Child, Preschool; Endocrinopathies; Female; Fundamental and applied biological sciences. Psychology; Growth Hormone - therapeutic use; Humans; Infant; Male; Medical sciences; Medicin och hälsovetenskap; Metabolic diseases; Obesity; Prader-Willi Syndrome - drug therapy; Psychomotor Disorders - drug therapy; Vertebrates: endocrinology; Endocrinopathy; Human; Somatotropin; Treatment; Adenohypophyseal hormone; Diseases of the osteoarticular system; Infant; Complex syndrome; Prader Labhart Willi syndrome; Endocrinology; Genetic disease
• ISSN: 0300-0664; 1365-2265; 1365-2265
• DOI: 10.1111/j.1365-2265.2007.03126.x

Summary Background  Prader–Willi syndrome (PWS) is a neurogenetic disorder characterized by muscular hypotonia, psychomotor delay, feeding difficulties and failure to thrive in infancy. GH treatment improves growth velocity and body composition. Research on the effects of GH on psychomotor development in infants with PWS is limited. Objective  To evaluate psychomotor development in PWS infants and toddlers during GH treatment compared to randomized controls. Design/patients  Forty‐three PWS infants were evaluated at baseline. Twenty‐nine of them were randomized into a GH group (n = 15) receiving 1 mg/m2/day GH or a non‐GH‐treated control group (n = 14). At baseline and after 12 months of follow‐up, analysis with Bayley Scales of Infant Development II (BSID‐II) was performed. Data were converted to percentage of expected development for age (%ed), and changes during follow‐up were calculated. Results  Infants in the GH group had a median age of 2·3 years [interquartile range (IQR) 1·7–3·0] and in the control group of 1·5 years (IQR 1·2–2·7) (P = 0·17). Both mental and motor development improved significantly during the first year of study in the GH group vs. the control group: median (IQR) change was +9·3% (–5·3 to 13·3) vs.–2·9% (–8·1 to 4·9) (P < 0·05) in mental development and +11·2% (–4·9 to 22·5) vs.–18·5% (–27·9 to 1·8) (P < 0·05) in motor development, respectively. Conclusion  One year of GH treatment significantly improved mental and motor development in PWS infants compared to randomized controls.