ISSN 1470-3947 (print) | ISSN 1479-6848 (online)

Endocrine Abstracts (2008) 16 S15.3

GH therapy in children with Prader Willi Syndrome (PWS)

Anita Hokken-Koelega

Sophia Children’s Hospital, Erasmus University Medical Center and Dutch Growth Research Foundation, Rotterdam, The Netherlands.

Background: PWS is neurogenetic disorder characterized by muscular hypotonia, hypogonadism, psychomotor delay, obesity, and short stature. Several reports have shown that GH treatment not only results in a remarkable growth, but also in an improvement of body composition and increased muscle strength and agility. Data about the effects of GH-treatment on motor and cognitive abilities and on respiratory parameters in PWS children are limited.

Methods: In a Dutch nation-wide study, 91 children with PWS (42 infants, 49 prepubertal children) were randomized to start with either GH treatment or no treatment for 1 or 2 years, resp. Age range was 0.6–11 years. Next to height, weight, head circumference and body composition by DXA scan, a complete polysomnography (PSG) was performed before and during the study. In addition, tests were performed to assess psychomotor development. Nocturnal sleep and respiration were recorded in a standard fashion. GH dosage was 1 mg/m2 per day.

Results: Median height SDS increased significantly during GH and did not change in controls. Median fat decreased significantly during GH and increased in controls. Median lean body mass increased significantly during GH and decreased in controls. The baseline Apnea Hypopnea Index (AHI) was 5.5 h (normal range 0–1 h), with very wide inter-individual variations. Of these, 2.9 h were identified as central apneas and 0.8 h were obstructive apneas. No correlation was found between AHI and age or BMI. During GH treatment, the AHI declined with ~50% in the majority of patients, mainly due to a reduction of central apneas. GH significantly improved mental and motor compared to randomized controls.

Conclusion: GH treatment normalizes height SDS and improves body composition in children with PWS. They have a high Apnea Hypopnea Index, mainly due to central apneas. During GH treatment a decline in AHI is found in the majority of patients, due to less central apneas. GH improves psychomotor development. Our results indicate that GH has an important role in PWS, also in young children.

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