Catherine S Choong

Charles F Verge

Elly Scheermeyer

Geoffrey Ambler

George Werther

Ian Hughes

Maria E Craig

Mark Harris

Patricia A Crock

Peter SW Davies

Phil Bergman

International Journal of Pediatric Endocrinology

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ORAL PRESENTATION Open Access
Response to low dose growth hormone
treatment in infants and toddlers with
Prader-Willi Syndrome
Elly Scheermeyer1*, Mark Harris2,3, Ian Hughes3, Patricia A Crock4,5, Geoffrey Ambler6,7, Charles F Verge8,9,
Phil Bergman10, George Werther11, Maria E Craig6,7, Catherine S Choong12,13, Peter SW Davies1
From 8th APPES Biennial Scientific Meeting
Darwin, Australia. 29 October – 1 November 2014
Aim
We aimed to assess the benefits and safety of low dose
growth hormone treatment (GHT, 4.5 mg/m2/week) in
young children with genetically confirmed Prader-Willi
Syndrome (PWS).
Methods
Data of 20 infants (2-12 months) and 24 toddlers (13-24
months) were collected from the PWS-OZGROW data-
base. The two groups were evaluated for standard devia-
tion scores (SDS) of height (length under age 2 years),
weight and BMI using the World Health Organization
standards (SDSWHO) and PWS specific BMI (SDSPWS),
bone age (BA), insulin-like growth factor-1 (IGF-1)
levels, hypotonia, developmental delay, spinal curvature,
sleep studies and adverse events over 2 years of GHT.
Results
At commencement of GHT infants had a reduced BMI
SDSWHO (P=0.003), while toddlers had a reduced height
SDSWHO (P=0.014). The height/length SDSWHO of infants
increased from -1.09±1.15 at baseline to -0.26±0.89 after
one year and -0.02±0.80 after two years GHT (GLM
repeated measures; P < 0.0001,) and in toddlers increased
from -2.11±1.45 to -1.11±1.11 and -0.87±0.94 (P <
0.0001). BMI SDSWHO increased in both groups (data not
shown), while BMI SDSPWS decreased (P < 0.0001, age
groups P > 0.05) from 0.40±0.84 to -0.07±0.67 at Year 1
and -0.31±0.95 at Year 2 (both age groups combined). Pre-
term and full term children did not differ significantly in
response to GHT, nor did children with deletion (14) and
uniparental disomy (16). All children had low to very low
serum IGF-1 at baseline which increased to within the
normal reference range for the majority of children (61%)
with the remainder modestly increased during the first
2 years of treatment. An improvement in tone, spinal cur-
vature and developmental delay was noted in those who
were more severely affected at baseline. Two children
developed scoliosis. Three children ceased GHT tempora-
rily to adjust positive airway pressure settings or for tonsil-
lectomy following onset or worsening of obstructive and/
or central sleep apnoea. Bone age was not advanced and
no other serious adverse events were reported during the
two year GHT.
Conclusion
Treating young children (<2 yrs) with PWS with 4.5 mg/m2/
week of GH normalises height and achieves IGF-1 levels in
the normal range in the majority of patients. The risk of
respiratory adverse events can be minimised by regular
monitoring. The dose was sufficient to keep height and
most IGF-1 values in the normal range and PWS specific
BMI SDS in a negative range and may lower potential risks
of long-term treatment of very young children with PWS.
On behalf of the PWS and OZGROW collaboration
Authors’ details
1Faculty of Medicine and Biomedical Sciences – School of Medicine, QCMRI,
The University of Queensland, Brisbane, QLD, Australia. 2Lady Cilento
Children’s Hospital, Brisbane, QLD, Australia. 3MRI-UQ, The University of
Queensland, Brisbane, QLD, Australia. 4John Hunter Children’s Hospital,
Newcastle, NSW, Australia. 5School of Medicine and Public Health, University
of Newcastle, Newcastle, NSW, Australia. 6The Children’s Hospital at
Westmead, Sydney, NSW, Australia. 7Discipline of Paediatrics and Child
Health, The University of Sydney, Sydney, NSW, Australia. 8Sydney Children’s
Hospital, Randwick, NSW, Australia. 9School of Women’s and Children’s
Health, University of New South Wales, Sydney, NSW, Australia. 10Monash
1Faculty of Medicine and Biomedical Sciences – School of Medicine, QCMRI,
The University of Queensland, Brisbane, QLD, Australia
Full list of author information is available at the end of the article
Scheermeyer et al. International Journal of Pediatric Endocrinology 2015, 2015(Suppl 1):O31
http://www.ijpeonline.com/content/2015/S1/O31
© 2015 Scheermeyer et al; licensee BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative
Commons Attribution License (http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and
reproduction in any medium, provided the original work is properly cited. The Creative Commons Public Domain Dedication waiver
(http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
Medical Centre, Melbourne, VIC, Australia. 11The Royal Children’s Hospital and
Murdoch Children’s Research Institute, Melbourne, VIC, Australia. 12Princess
Margaret Hospital Perth, Perth, WA, Australia. 13School of Pediatrics and Child
Health, University of Western Australia, Perth, WA, Australia.
Published: 28 April 2015
doi:10.1186/1687-9856-2015-S1-O31
Cite this article as: Scheermeyer et al.: Response to low dose growth
hormone treatment in infants and toddlers with Prader-Willi Syndrome.
International Journal of Pediatric Endocrinology 2015 2015(Suppl 1):O31.
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Scheermeyer et al. International Journal of Pediatric Endocrinology 2015, 2015(Suppl 1):O31
http://www.ijpeonline.com/content/2015/S1/O31
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Response to low dose growth hormone treatment in infants and toddlers with Prader-Willi Syndrome

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Response to low dose growth hormone treatment in infants and toddlers with Prader-Willi Syndrome

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