ESPE Abstracts (2019) 92 P2-168

Adherence and Long-Term Outcomes of Therapy in Pediatric Subjects in Slovakia using Easypod™ Electromechanical Device for Growth Hormone Treatment: The Phase IV Multicentre Easypod™ Connect Observational Study (ECOS) Ľudmila Koštálová, Svetlana Bieliková, Miriam Ciljaková, Adriana Dankovcíková, Juliana Ferenczová, Slavomíra Kyšková, Eva Mendelová, Zuzana Pribilincová, Vilja Šandriková, Lubica Tichá, Marcela Balošáková

Ludmila Kostalova1, Svetlana Bielikova2, Miriam Ciljakova3, Adriana Dankovcikova4, Juliana Ferenczova4, Slavomira Kyskova5, Eva Mendelova6, Zuzana Pribilincova1, Vilja Sandrikova7, Lubica Ticha1, Marcela Balosakova8

1Department of Pediatric, National Institute of Child´s Diseases, Bratislava, Slovakia. 2Endokrino, Banska Bystrica, Slovakia. 33Pediatric Department of National Institute of Endocrinology and Diabetology, Lubochna, Slovakia. 4I. Children and Adolescents Clinic, Children´s Faculty Hospital, Kosice, Slovakia. 5Ambulance for Children and Adolescents, Pediatric Endocrinology, Zilina, Slovakia. 6Pediatric Department of National Institute of Endocrinology and Diabetology, Lubochna, Slovakia. 7Endokid, Prievidza, Slovakia. 8MERCK, Bratislava, Slovakia

The global Easypod™ Connect Observational Study (ECOS) reported that adherence to recombinant human growth hormone (r-hGH; Saizen®) was maintained ~80% for up to 3 years. We now report the adherence outcomes in the cohort of ECOS patients in Slovakia (NCT01400984).

Patients aged 3–16 years treated with r-hGH administered via easypod™ from ≥4 months to 4 years and with a documented start date were included. Good adherence (proportion of days with injection received/days with injections planned) was defined as ≥80%. Secondary outcomes included change in height standard deviation score (SDS), change in height velocity SDS and trends between adherence and growth outcomes. Data on adherence were downloaded from the easypod™ device; demographic, auxological and diagnostic data were taken from medical notes. All analyses were descriptive.

Ninety-two r-hGH-naive patients were included (median age 9 years; 39% female, 61% male; 36 had growth hormone deficiency [GHD], 48 were small for gestational age [SGA] and eight had Turner syndrome). Data from 80 patients with adherence data for ≥3 months after starting treatment were analysed. Median (Q1;Q3) treatment duration was 845 (542;1063) days and the median starting dose was 0.0318 (0.028;0.034) mg/kg. Overall median adherence was 92.35%. When analysed by 1-year intervals, median adherence was 95.5% to 1 year, 93% to 2 years and 83% to 3 years. After 1 year, the overall change in growth outcomes was clinically meaningful: median change in height SDS was 0.49 (0.37;0.62) and median change in height velocity SDS was 2.53 (1.32;3.87). Analysis of growth outcomes at 1 year in patients who had no missing data and no gaps in treatment >1 week (complete analysis set [CAS]; N=52) produced similar results: overall median adherence was 93%, median change in height SDS 0.49 (0.37;0.62) and change in median height velocity SDS 2.54 (1.54;3.73). Up to 1 year the Spearman product-moment correlation between adherence and change in height SDS was positive and significant (P=0.0143) in the subset of patients with GHD.

Overall median adherence to treatment up to 3 years was >90% in this cohort of r-hGH-naïve patients. Growth outcomes were clinically meaningful and similar among all patients, and a positive association was seen between adherence and growth outcomes at 1 year for patients with GHD.

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