ESPE Abstracts (2019) 92 P2-167

Adherence and Long-Term Outcomes of Therapy in Pediatric Subjects in Argentina using Easypod™ Electromechanical Device for Growth Hormone Treatment: The Phase IV Multicentre Easypod™ Connect Observational Study (ECOS)

Cinthia D Chareca1, Alicia Belgorosky2, Javier Chiarpenello3, Lia Colombi4, Titania Pasqualini5, Cristian Von Schulz Hausmann1, Aria Assefi1, mirta miras6

1Merck S.A (Argentina), an affiliate of Merck KGaA, darmstadt, Germany, Ciudad de Buenos Aires, Argentina. 2Hospital de Pediatría Garrahan, Ciudad de Buenos Aires, Argentina. 3Hospital Centenario de Rosario, Rosario, Argentina. 4Hospital Humberto Notti, Mendoza, Argentina. 5Hospital italiano Buenos Aires, Ciudad de Buenos Aires, Argentina. 6Hospital de niños santísima trinidad, Cordoba, Argentina

The Easypod™ Connect Observational Study (ECOS) assessed real-time adherence in patients from 24 countries who were receiving recombinant human growth hormone (r-hGH; Saizen®) via easypod™, which is an electronic injection device. Overall, ECOS showed mean adherence was maintained at ~80% for up to 3 years. Here, we assess the adherence to r-hGH administered via easypod™ in the Argentinian cohort of patients from ECOS (NCT01582334).

Patients aged 2–18 years treated with r-hGH administered via easypod™ for ≥6 months and ≥5 years, with a documented start date were eligible. Good adherence (proportion of days with injection received/days with injections planned) was defined as ≥80%. Growth outcomes (change in height standard deviation score [SDS] and change in height velocity SDS) and trends between adherence and growth outcomes were secondary outcomes. Adherence data were downloaded from the easypod™ device; demographic, auxological and diagnostic data were taken from medical notes. All analyses were descriptive.

Sixty-eight patients overall were included in the observational multicenter study (median age 11 years, 71% male and 29% female); 63 patients with adherence data for the 3 months after starting treatment were included in the easypod™ adherence data analysis set. The patient diagnoses were: 44 had growth hormone deficiency (GHD), 11 were small for gestational age (SGA) and eight had Turner syndrome (TS); 33 were growth hormone naïve. After 1 year of treatment, median (Q1:Q3) adherence in the easypod™ adherence data analysis set (N=49) was 88.5% (67.9%:95.6%). Good adherence decreased each year but was maintained to 3 years: 81.0% (53.2%:93.5%); N=17. After 1 year, the median (Q1;Q3) change in height SDS was 0.43 (0.21;0.64) and the change in height velocity SDS was 2.02 (0.33;4.30). Sub-analysis of adherence and growth outcomes at 1 year in patients with no missing data and no gaps in treatment >1 week (N=22) produced similar results: change in height SDS was 0.45 (0.21;0.64) and change in height velocity SDS was 2.15 (0.61;4.30). One-year growth outcomes in r-hGH treatment-naïve patients showed variable outcomes based on the origin of GHD. There was a positive trend between adherence and change in height SDS at 1 year for patients with GHD, although the number of patients was small (n=44).

In agreement with the results from the global analyses of ECOS, treatment with r-hGH administered via easypod™ led to high adherence rates in this representative population from Argentina. Overall, 1-year growth outcomes were clinically meaningful.