BSPED2012 Poster Presentations (1) (66 abstracts)
Key efficacy issues in the use of recombinant human GH in children with prader–willi syndrome
Mary Thornton 1 , Indi Banerjee 2 , Raja Padidela 2 , Elaine O’Shea 2 , Julie Jones 2 , Rakesh Amin 2 , Leena Patel 2 , Sarah Ehtisham 2 , Peter Clayton 3 & Mars Skae 2
1University of Manchester, Medical School, Manchester, UK; 2Department of Paediatric Endocrinology, Royal Manchester Children’s Hospital, Manchester, UK; 3Endocrine Science Research Group, University of Manchester, Manchester, UK.
Prader-Willi syndrome (PWS) is a rare genetic condition characterised by hypotonia, early feeding difficulties, hyperphagic obesity, hypogonadism and short stature; with an incidence between 1/15 000 and 1/25 000 live births in the UK. It is caused by failed expression of paternally inherited genes in the imprinting region of chromosome 15q11.2–q13. Recombinant human GH (rhGH) is the main pharmacological treatment used in PWS.
Aims: We aimed to review the use of rhGH in our cohort against current UK National Institute of Health and Clinical Excellence (NICE) recommendations to determine effects on growth outcomes and side effects in patients.
Methods: A retrospective audit of case notes was performed in 30 paediatric PWS patients treated with rhGH at our centre, to analyse monitoring standards, frequency of side effects and auxology outcomes. Results: In our cohort, 37% had biochemical evidence of GH deficiency (GHD) and 93% of our cohort was treated with a lower median dose of rhGH (mdGH) (24.0 μg/kg per day 16.5–37.0) throughout their treatment period than NICE recommendations for weight (35.0 μg/kg per day). No significant correlation was found between mdGH and change in height SDS or BMI SDS and mean final heights attained in our patients were comparable to the literature (Takeda et al., 2009). 20% of patients developed impaired glucose tolerance prior to or on treatment and fasting glucose and insulin levels were not predictive of glycaemic impairment when compared with oral glucose tolerance testing (OGTT). Scoliosis had a high prevalence in our patients (57%) and 40% worsened on rhGH.
Conclusions: Lower rhGH treatment doses may not significantly impair growth and metabolic composition and demonstrate better cost benefit than current NICE recommendations in PWS patients. We recommend that OGTT be used for effective glycaemic monitoring, whilst regular spinal assessment is essential.
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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