Response to low dose growth hormone treatment in infants and toddlers with Prader-Willi Syndrome
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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 database. The two groups were evaluated for standard deviation 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). Preterm and full term children did not differ significantly in response to GHT, nor did children with deletion (14) and Faculty 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
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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 curvature and developmental delay was noted in those who were more severely affected at baseline. Two children developed scoliosis. Three children ceased GHT temporarily to adjust positive airway pressure settings or for tonsillectomy 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 (
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