ESPE Abstracts (2018) 89 P-P2-255

Clinical and Cost-Effectiveness of GH Treatment for Children in Wales

Raluca-Monica Popa, Justin T. Warnerb & John W. Gregoryc

aResearch Methodology Department, University of Medicine and Pharmacy, Tirgu Mures, Romania; bDepartment of Child Health University Hospital of Wales, Cardiff, UK; cDivision of Population Medicine, School of Medicine, Cardiff University, Cardiff, UK

Background: GH treatment has been used for the last 30 years for children with short stature with varying individual responses.

Objective: Analysis of final height SDS (standard deviation score) and the factors influencing it in children treated with growth hormone.

Material and methods: Subjects across Wales who received GH treatment, part supervised by tertiary center staff and reached final height while on treatment, were identified by database searching. Final height was defined when the switch to adult dosing occurred or a height velocity (HV) <1 cm/year. Exclusion criteria included subjects on GH treatment for less than a year, a HV <1 cm/year on GH or GH started after growth completion. Variables analyzed included age, sex, diagnosis, presence of concomitant hypothyroidism, adrenal failure, age at GH initiation, height SDS at diagnosis, number of years on treatment, puberty induction, mid-parental height SDS, age at GH cessation, mean GH dose throughout treatment, height gain at 1 year, total height gain, cost of treatment (£/cm gained). SPSS v. 17.0 was used for statistical analysis, with a level of significance of α=0.05.

Results: 141 subjects were identified with a sex ratio of F:M of 1.2:1. 101 (71.6%) had GH deficiency (GHD) from various causes, 26 (18.4%) had Turner syndrome (TS) and 14 (9.9%) had other diagnoses (Prader-Willi Syndrome, constitutional delay in growth and puberty, small for gestational age, renal disorders, idiopathic short stature). Mean age at treatment initiation was 10.4±3.3 years for the whole sample. The median period on GH treatment was 5.1years. 71 (50.3%) subjects required puberty induction. Total height gain was 0.87SD for GHD, 0.09SD for TS and 0.74SD for the other diagnoses (P=0.043) with a cost of £6323.8/cm for GHD, £8465.9/cm for TS and £4272.6/cm for other diagnoses (P=0.045). 56 (53.3%) children reached a final height within mid-parental height range. Best predictors for response to treatment were height SD at diagnosis, HV in the first year, years on treatment, and age at treatment start. The mean final height for the group with Turner syndrome was 148.7±6.1 cm.

Conclusions: GH treatment recommendation in Wales follows current guidelines. The response to treatment is variable, with a significant proportion of subjects not reaching the predicted final height. Turner syndrome benefits the least from GH therapy.

Keywords: final height, GH treatment, audit

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