Controlled growth hormone study in children with Prader Willi Syndrome.

Summary of the Dutch National Growth Hormone Study in Children with Prader Willi Syndrome.

Title:

Multicenter, randomized, controlled growth hormone study in children with Prader Willi Syndrome: effects on growth, body composition, activity level and psychosocial development” MEC 2001/230.

Short title:

Controlled growth hormone study in children with Prader Willi Syndrome.

Background:

Children with Prader Willi Syndrome have often short stature and have an abnormal body composition (Increased fatpercentage and decreased lean body mass). Physical activity level is therefore decreased in children with Prader Willi Syndrome. Children with Prader Willi syndrome often have mental retardation and behavioral problems.

Recent studies showed an improvement in height and body composition during growth hormone (GH) treatment in children with Prader Willi Syndrome. Preliminary data showed also an improvement in activity level, metabolism, respiratory function, behaviour and social-emotional development.

Aim:

To study the effects of GH-treatment versus no GH treatment in children with Prader Willi Syndrome on changes in height, weight, body composition, muscle strength, activity level, psychosocial development, metabolism and respiratory function.

Patients:

85 children with PWS, aged 6 mo. to 16 years at start of the study.

Intervention:

Treatment with GH: Genotropin ® 1mg/m2/d s.c.

Design:

Children will be randomly divided into 3 subgroups: infants, prepubertal children and adolescents. Infants and prepubertal children will be treated in a controlled design. (Start GH-treatment at start of the study or after 1 or two years resp.) Adolescents will be treated in a non-controlled design. All children over the age of 3 will receive dietary advice and an exercise program.

Anthropometric assessments will be performed every 3 months.

Yearly assessment of body composition (DEXA), metabolism (Indirect calorimetry), muscle strength (quadriceps dynamometry) and activity level (Activity monitoring) will be performed.

Cognition (subtests of WISC, WPPSI or BOS), behaviour (DBC), social emotional development (VABS) and quality of life (DUX 25) will be measured yearly.

In a subgroup of children pulmonary CO2 responsiveness will be measured.

Objectives:

Primary:

To asses effects of GH-treatment vs. no GH-treatment in children with PWS on
Height, weight, body composition
Muscle mass, muscle strength and daily life activity
Cognition, behaviour and social emotional development
Resting Energy Expenditure
CO2 responsiveness.

Secondary:

To study the effect of additional dietary advise and physical exercise on body composition in children with PWS treated with GH vs, not treated with GH.

GH treatment improves height, weight, body composition, muscle strength, activity level, psychosocial development, psychomotor development in infants, metabolism and respiratory function versus no GH treatment in children with Prader Willi Syndrome.

N/A

Treatment with GH: Genotropin ® 1mg/m2/d s.c. vs. no GH-treatment.

Dietary and exercise advice.