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Endocrine Abstracts (2016) 45 OC6.7 | DOI: 10.1530/endoabs.45.OC6.7

BSPED2016 Oral Communications Oral Communications 6- Endocrine (9 abstracts)

Early treatment with rhGH in patients with Prader-Willi syndrome results in improved height with no respiratory adverse effects

Kyriaki Alatzoglou 1 , Mahalakshmi Gopalakrishnamoorthy 1 , Emily Trewella 1 , Aayesha Mulla 1 , Hui-Leng Tan 2 & Nicola Bridges 1

1Department of Paediatric Endocrinology and Diabetes, Chelsea and Westminster Hospital NHS Foundation Trust, London, UK; 2Department of Respiratory Medicine, Royal Brompton Hospital NHS Foundation Trust, London, UK.

Background: Prader-Willi syndrome (PWS) is a complex genetic disorder caused by lack of expression of paternally inherited imprinted genes on Chr15q11-q13. rhGH has beneficial effects on growth, body composition and development. Starting age, dose titration and monitoring remain controversial.

Objective: To study retrospectively children who presented in our multidisciplinary PWS clinic and assess response to rhGH treatment in terms of auxology, IGF1 concentration and potential complications.

Method & Patients: In total of 47 patients (male 27, female 20) were followed up; 5/47 were lost to follow-up and 2/47 refused rhGH treatment. 40 patients were treated with rhGH, all had detailed sleep studies before, and six weeks after starting treatment.

Results: Treatment started at a mean age of 2.1±2.6 years (range 0.58–12.8), 45% of patients (n=18) started rhGH before the age of one (0.58–0.97 years). 15% (n=6) had evidence of sleep apnoea, requiring non invasive ventilation before starting rhGH. Mean starting rhGH dose was 0.025 mg/kg/day (0.5 mg/m2/day) increased to 1 mg/m2/day following the second sleep study. At one year, mean dose was 0.7±0.2 mg/m2/day. Pre-treatment mean height was −1.65±1.1 SDS (−4.47 to 0.37 SDS), with a weight of −1.43±1.8 SDS (−5.5 to 3.3) and BMI of −0.46±1.6 SDS (−3 to 4). After one year there was an increase of 1.37SDS in Ht (mean −0.28±0.94, range −2.4 to +2.3), with a mean weight of −0.24±1.8 SDS (range −4.0 to 3.7) and BMI of 0.08±2 SDS (−3.0 to 4.6). No patient had worsening respiratory status and 2/6 patients discontinued ventilatory support. One patient paused treatment during spinal surgery and restarted afterwards. IGF1 was >+2.0 SDS in 60% of patients and the dose of rhGH remained unchanged with repeat yearly IGF1 measurement.

Conclusion: Early treatment with GH results in improved height in the first year with no adverse effect on respiratory function. We recommend dose titration using auxology and IGF1 concentration.

Volume 45

44th Meeting of the British Society for Paediatric Endocrinology and Diabetes

British Society for Paediatric Endocrinology and Diabetes 

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