Adherence to treatment in children with growth hormone deficiency, small for gestational age and Turner syndrome in Mexico: results of the Easypod? connect observational study (ECOS)

摘要

Background Assessing adherence to growth hormone (GH) is challenging. The Easypod? connect device delivers preset doses of recombinant human GH (r-hGH) and stores a digital record of adherence that can be shared with healthcare provider. We assessed adherence to r-hGH delivered with Easypod? according to the approved pediatric indications for r-hGH: growth hormone deficiency (GHD), born small for gestational age (SGA) who failed to show catch-up growth and Turner syndrome (TS). Methods ECOS (NCT01555528) was a multicenter (24 countries), 5-year, longitudinal, observational study, which aimed to evaluate country-specific adherence to r-hGH therapy prescribed via the Easypod? electronic injection device. The primary endpoint was yearly adherence. Secondary endpoints were height velocity, height velocity standard deviation scores (SDS), height, height SDS and IGF-1 concentrations. Clinical and auxological data were obtained from medical records and adherence from Easypod? logs. Results This study included 147 Easypod?-naive Mexican children assessed during 3 years (mean age: 9.96 ±3.41 years, 56.8% boys, mean height SDS at baseline: - 2.17±0.97): 118 with GHD, 24 SGA and 5 with TS. A total of 105 (71.4%) patients were GH naive. Overall median adherence was >90% over the first year of treatment and >80% at 3 years. Adherence was not different by r-hGH indication or between GH-nai've or experienced patients. At 1-year follow-up, mean change in height SDS was 0.57 ±0.34, whereas mean height velocity SDS was 2.85 ±2.51. In all, 84.7% patients had normal IGF-1 concentrations at 1-year follow-up. Adherence was associated with change in height SDS (r = 0.239, p = 0.005) and height velocity SDS (r = 0.194, p = 0.027). Conclusion Adherence rates with the Easypod? device are high and maintained over time in GHD, SGA and TS Easypod?-naive Mexican patients. High adherence is associated with better outcomes. Easypod? assists physicians in monitoring adherence to r-hGH.