Response to growth hormone treatment in Prader-Willi syndrome: Auxological criteria versus genetic diagnosis

Elly Scheermeyer, Ian Paul Hughes, Mark F. Harris, Geoff Ambler, Patricia Crock, Charles F. Verge, Maria E. Craig, Phil Bergman, George Werther, Mieke Van Driel, Peter S. W. Davies, Catherine S Y Choong

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Abstract

Aim 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. Methods 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. Results 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. Conclusions 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.

Original languageEnglish
Pages (from-to)1045-1051
Number of pages7
JournalJournal of Paediatrics and Child Health
Volume49
Issue number12
DOIs
Publication statusPublished - Dec 2013

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Prader-Willi Syndrome
Growth Hormone
Therapeutics
Body Mass Index
Bone and Bones

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Scheermeyer, E., Hughes, I. P., Harris, M. F., Ambler, G., Crock, P., Verge, C. F., ... Choong, C. S. Y. (2013). Response to growth hormone treatment in Prader-Willi syndrome: Auxological criteria versus genetic diagnosis. Journal of Paediatrics and Child Health, 49(12), 1045-1051. https://doi.org/10.1111/jpc.12294
Scheermeyer, Elly ; Hughes, Ian Paul ; Harris, Mark F. ; Ambler, Geoff ; Crock, Patricia ; Verge, Charles F. ; Craig, Maria E. ; Bergman, Phil ; Werther, George ; Van Driel, Mieke ; Davies, Peter S. W. ; Choong, Catherine S Y. / Response to growth hormone treatment in Prader-Willi syndrome : Auxological criteria versus genetic diagnosis. In: Journal of Paediatrics and Child Health. 2013 ; Vol. 49, No. 12. pp. 1045-1051.
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title = "Response to growth hormone treatment in Prader-Willi syndrome: Auxological criteria versus genetic diagnosis",
abstract = "Aim 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. Methods 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. Results 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. Conclusions 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.",
author = "Elly Scheermeyer and Hughes, {Ian Paul} and Harris, {Mark F.} and Geoff Ambler and Patricia Crock and Verge, {Charles F.} and Craig, {Maria E.} and Phil Bergman and George Werther and {Van Driel}, Mieke and Davies, {Peter S. W.} and Choong, {Catherine S Y}",
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Scheermeyer, E, Hughes, IP, Harris, MF, Ambler, G, Crock, P, Verge, CF, Craig, ME, Bergman, P, Werther, G, Van Driel, M, Davies, PSW & Choong, CSY 2013, 'Response to growth hormone treatment in Prader-Willi syndrome: Auxological criteria versus genetic diagnosis' Journal of Paediatrics and Child Health, vol. 49, no. 12, pp. 1045-1051. https://doi.org/10.1111/jpc.12294

Response to growth hormone treatment in Prader-Willi syndrome : Auxological criteria versus genetic diagnosis. / Scheermeyer, Elly; Hughes, Ian Paul; Harris, Mark F.; Ambler, Geoff; Crock, Patricia; Verge, Charles F.; Craig, Maria E.; Bergman, Phil; Werther, George; Van Driel, Mieke; Davies, Peter S. W.; Choong, Catherine S Y.

In: Journal of Paediatrics and Child Health, Vol. 49, No. 12, 12.2013, p. 1045-1051.

Research output: Contribution to journalArticleResearchpeer-review

TY - JOUR

T1 - Response to growth hormone treatment in Prader-Willi syndrome

T2 - Auxological criteria versus genetic diagnosis

AU - Scheermeyer, Elly

AU - Hughes, Ian Paul

AU - Harris, Mark F.

AU - Ambler, Geoff

AU - Crock, Patricia

AU - Verge, Charles F.

AU - Craig, Maria E.

AU - Bergman, Phil

AU - Werther, George

AU - Van Driel, Mieke

AU - Davies, Peter S. W.

AU - Choong, Catherine S Y

PY - 2013/12

Y1 - 2013/12

N2 - Aim 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. Methods 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. Results 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. Conclusions 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.

AB - Aim 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. Methods 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. Results 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. Conclusions 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.

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