Endocrine Abstracts

Introduction: The easypod™ Connect Observational Study (ECOS) was a global study with easypod™, an electronic injection device for recombinant human growth hormone (r-hGH; Saizen^®). This study aimed to assess adherence and growth outcomes of patients treated with r-hGH via easypod™ in the UK-based ECOS cohort (NCT01263457).

Methods: Patients aged 2–18 years, with ≥3 months of easypod™ data after enrolment into the study comprised the ECOS data analysis set (DAS); ECOS DAS patients with ≥3 months of easypod™ data after initiating easypod™ use comprised the easypod™ DAS. The primary objective was to assess adherence; growth outcomes (Δheight [ΔHt], ΔHt standard deviation score [ΔHtSDS], height velocity [HV], HVSDS), correlation between adherence and growth outcomes (Spearman’s product moment), and the impact of adherence on IGF-I were secondary objectives. All analyses were descriptive.

Results: The study included 63 patients; growth hormone deficiency (n=44), small for gestational age (n=8), Turner syndrome (n=7) and other diagnoses (n=4). Median age was 12 years with 21 (33%) female. In the ECOS DAS, median adherence was 92.75% over 1 year (n=56), 89.70% over 2 years (n=35) and 71.90% over 3 years (n=13). After 1 year, median results showed ΔHt 7.6 cm, ΔHtSDS 0.42, HV 7.69 cm/year and HVSDS 2.36. Significant correlations were detected between adherence and ΔHtSDS (n=44; P=0.018) and HVSDS (n=43; P=0.006) in the ECOS DAS after 1 year. In GH-naïve patients from the easypod™ DAS (n=42/63; 67%), median adherence was 93.3% over 1 year (n=40), 92.7% over 2 years (n=35) and 77.8% over 3 years (n=22); after 1 year, median growth outcomes were ΔHt 8.25 cm, ΔHtSDS 0.46, HV 8.55 cm/year and HVSDS 2.01. IGF-I concentrations were normal after 1 year in 17/22 patients with data [77.3%]); 3 (13.6%) had abnormally low and 2 (9.1%) had abnormally high concentrations.

Conclusions: Treatment with r-hGH via easypod™ led to high adherence in this UK ECOS population although slightly lower than in the global analysis. After 1 year, treatment efficacy, IGF-I normalisation and significant correlations between adherence and ΔHtSDS and HVSDS were reported.