ESPE Abstracts (2016) 86 P-P2-656

Improved Growth Outcomes with Jet Delivery of Growth Hormone in Children are Maintained Over Long-Term Treatment

Maria Michaelidoud, Alastair D Knightc, Sue Whittenb, Priti Bajajb & Helen A Spoudeasa

aLondon Centre for Paediatric Endocrinology, Great Ormond Street and University College London Hospitals, London, UK; bFerring Pharmaceuticals, London, UK; cEvicom Ltd, Twickenham, UK; dNottingham University Hospitals NHS Trust, Nottingham, UK

Background: We previously reported in a national cohort, that adherence to subcutaneous Growth Hormone (GH) treatment in children is better with jet delivery compared with needle devices (Spoudeas et al. Patient Prefer Adherence. 2014;8:1255–1263). We also separately reported that adherent children showed significantly improved height outcomes at one year.

Objective and hypotheses: To examine the potential influence of adherence and demographic covariates on growth outcomes of children using Growth Hormone (GH) jet delivery beyond one year of treatment.

Method: A cohort of children aged <16 years treated with GH via jet delivery was identified at a split-site centre (GOSH and UCLH, UK). Adherence and growth parameters were followed-up over a treatment period beyond one year. Adherence was evaluated using the Proportion of Days Covered (PDC) index. Patients with a PDC score >0.8 were considered adherent. Standard deviation scores for Height (HTSDS) and Height Velocity (HVSDS) were primary outcomes compared to pre-treatment and target height. The influence of categorical demographics before treatment was assessed.

Results: From a cohort of 72 patients, 31 (20M, 11F) were defined as adherent at one year and followed-up for median (range) 3.12 (1.46–4.85) years. Mean±S.D. HTSDS and HVSDS were significantly improved from baseline at one year and assessment end (−2.03±1.18, −1.34±1.22, −1.02±1.21 and −1.10±2.51, 2.56±2.51, 0.74±3.91, P<0.001). HTSDS and HVSDS outcomes at end of treatment were not influenced by thyroxine use, previous irradiation, years before start of treatment, sex and age at onset.

Conclusion: Jet delivery of GH for a treatment period beyond one year provides children with improved height outcomes which are independent of demographics pre-treatment. High persistence of use with GH jet delivery, as reported earlier, may contribute to these long-term outcome benefits.

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