Conflict of interest: None declared.
Response to growth hormone treatment in Prader–Willi syndrome: Auxological criteria versus genetic diagnosis
Article first published online: 19 JUN 2013
© 2013 The Authors. Journal of Paediatrics and Child Health © 2013 Paediatrics and Child Health Division (Royal Australasian College of Physicians)
Journal of Paediatrics and Child Health
Volume 49, Issue 12, pages 1045–1051, December 2013
How to Cite
Scheermeyer, E., Hughes, I., Harris, M., Ambler, G., Crock, P., Verge, C. F., Craig, M. E., Bergman, P., Werther, G., van Driel, M., Davies, P. S. and Choong, C. S. (2013), Response to growth hormone treatment in Prader–Willi syndrome: Auxological criteria versus genetic diagnosis. Journal of Paediatrics and Child Health, 49: 1045–1051. doi: 10.1111/jpc.12294
- Issue published online: 10 DEC 2013
- Article first published online: 19 JUN 2013
- Manuscript Accepted: 15 APR 2013
- Australasian Paediatric Endocrine Group
- growth hormone;
- Prader–Willi syndrome
The Australian Prader-Willi Syndrome (PWS) database was established to monitor the efficacy and safety of growth hormone (GH) treatment in PWS. This study aims to compare response to GH based on eligibility criteria.
Comparative study: 72 children received GH on the basis of short stature or evidence of GH deficiency (pre-2009: PWS-SS) and 94 on a genetic diagnosis (post-2009: PWS-Dx). We report on mandatory patient data for GH prescription: median and standard deviation score (SDS) for height and body mass index (BMI), waist/height ratio, bone age/chronological age ratio and adverse events. Comparisons were made using non-parametric tests.
At baseline, the PWS-SS cohort was shorter (height SDS: –2.6 vs. –1.1, P < 0.001), had a lower BMI (0.6 vs. 1.5 SDS, P < 0.05) and greater bone age delay (bone age/chronological age: 0.7 vs. 0.9, P < 0.05) than the PWS-Dx cohort. PWS-SS parents were shorter (mid-parental height SDS: −0.13 vs. 0.28, P < 0.005). Mean change in height over 2 years was 0.9 SDS and in BMI using PWS reference standards –0.3 SDSPWS (n = 106) (year 2, height SDS: PWS-SS = –1.7, PWS-Dx = 0.1; BMI SDSPWS: PWS-SS = –1.0, PWS-Dx = –0.6). The waist/height ratio reduced (PWS-Dx: 0.60 vs. 0.56, P < 0.05) and bone age delay was unchanged over this period. No serious adverse events were reported.
The PWS-SS cohort represents a subgroup of the wider PWS-Dx population; however both cohorts improved height SDS with normalisation of height in the PWS-Dx cohort and lowering of BMI relative to PWS standards supporting the efficacy of treatment under the current Australian GH programme.