Elsevier

The Journal of Pediatrics

Volume 145, Issue 6, December 2004, Pages 744-749
The Journal of Pediatrics

Original Article
Growth hormone improves mobility and body composition in infants and toddlers with Prader-Willi syndrome

https://doi.org/10.1016/j.jpeds.2004.08.002Get rights and content

Objectives

To determine the effect of growth hormone (GH) on body composition and motor development in infants and toddlers with Prader-Willi syndrome (PWS).

Study design

Twenty-nine subjects with PWS (4-37 months of age) were randomized to GH treatment (1mg/m2/day) or observation for 12 months. Percent body fat, lean body mass, and bone mineral density were measured by dual x-ray absorptiometry; energy expenditure was measured by deuterium dilution; and motor constructs of mobility (M) and stability (S) were assessed using the Toddler Infant Motor Evaluation (TIME).

Results

GH-treated subjects, compared with controls, demonstrated decreased percent body fat (mean, 22.6% ± 8.9% vs 28.5% ± 7.9%; P < .001), increased lean body mass (mean, 9.82 ± 1.9 kg vs 6.3 ± 1.9 kg; P < .001), and increased height velocity Z scores (mean, 5. 0 ± 1.8 vs 1.4 ± 1.0; P < .001). Patients who began GH before 18 months of age showed higher mobility skill acquisition compared with controls within the same age range (mean increase in raw score, 284 ± 105 vs 206 ± 63; P < .05).

Conclusions

GH treatment of infants and toddlers with PWS for 12 months significantly improves body composition and when begun before 18 months of age increases mobility skill acquisition. These results suggest that GH therapy instituted early in life may lessen deterioration of body composition in PWS while also accelerating motor development.

Section snippets

Patients and methods

Twenty-nine infants and toddlers with PWS 4 to 37 months of age, (mean age 15 ± 9 months; 13 females) were enrolled in the study after informed consent was obtained from a parent or legal guardian. All patients had a diagnosis of PWS confirmed by high-resolution cytogenetics, fluorescent in situ hybridization, and/or methylation studies. Seventeen subjects had deletion of chromosome 15q11-13, 11 had uniparental disomy, and 1 patient was diagnosed by an abnormal methylation test. Length/height

Results

Patient characteristics and study results are summarized in Table I, Table II, Table III and are presented as mean ± SD.

Discussion

Infants and toddlers with PWS commonly demonstrate hypotonia, associated with poor suck, poor feeding, compromised respiratory function, early failure to thrive, and delay in attainment of developmental motor skills. Body fat measurements are increased even in underweight infants with PWS.7., 8., 5., 33. Early abnormalities in body composition in PWS, therefore, are present before the onset of characteristic hyperphagia and progressive obesity, and they are qualitatively similar to those

References (33)

  • P. Burman et al.

    Endocrine dysfunction in Prader-Willi syndrome: a review with special reference to GH

    Endocrinol Rev

    (2001)
  • R.D. Nicholls et al.

    Genetic abnormalities in Prader-Willi syndrome and lessons from mouse models

    Acta Paediatrica Scand

    (1999)
  • V.A. Holm et al.

    Prader-Willi Syndrome: consensus diagnostic criteria

    Pediatrics

    (1993)
  • E.M. Ritzen et al.

    Growth hormone treatment of patients with Prader-Willi syndrome. Swedish Growth Hormone Advisory Group

    J Pediatr Endocrinol

    (1999)
  • A.L. Carrel et al.

    Benefits of long-term GH therapy in Prader-Willi syndrome: a 4-year study

    J Clin Endocrinol Metab

    (2002)
  • L.J. Miller et al.

    Sequence comparison methodology for the analysis of movement patterns in infants and toddlers with and without motor delays

    Am J Occup Ther

    (1993)
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    Supported by grants from NIH M01 RR33186 and Pharmacia Inc. (Pfizer).

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