ESPE Abstracts (2019) 92 P1-220

Real-World Data From Electronic Monitoring of Adherence to Growth Hormone Treatment in Children with Growth Disorders: A Descriptive Analysis

Ekaterina Koledova1, Vincenzo Tornincasa2, Paula van Dommelen3


1Merck Healthcare KGaA, Darmstadt, Germany. 2Ares Trading S.A., 1267 Coinsins, Switzerland. 3The Netherlands Organization for Applied Scientific Research TNO, Leiden, Netherlands


Poor adherence to long-term growth hormone (GH) treatment can lead to suboptimal clinical outcomes. The easypod™ connect eHealth platform enables healthcare professionals to obtain an accurate picture of real-world adherence by allowing patients to transmit adherence data to a database. Our aims were to assess adherence to r-hGH (Saizen, Merck KGaA, Darmstadt, Germany) treatment with the easypod connect platform in children from treatment start to 48 months and to investigate the effect of age and sex on adherence and the association between transmission frequency and adherence.

Patients transmitting >10 injections between January 2007 and February 2019 were analyzed. Adherence, calculated as mg injected/mg prescribed (dosage and settings defined by the care team) and categorized as high (≥85%), intermediate (>56%–84%) or low (≤56%), was assessed at months 1, 3, 6, 12, 24, 36 and 48 and then explored by puberty status (cut-offs at 10 years for girls and 12 years for boys) and sex. We calculated the transmission ratio (total number of transmissions versus the total number of recorded days of easypod connect use) as a measure of patient engagement in disease management.

Longitudinal records were available for 13,553 patients. Overall, more patients had high adherence (n=9,578 [71%]) than medium (n=2,989 [22%]) or low (n=986 [7%]). Although the proportion of patients in the high adherence category decreased over time (from 87% at month 1 to 65% at month 48), at each time point more patients were in the high adherence category than in the medium/low adherence categories. Overall, a slightly higher proportion of girls had high adherence compared with boys (72% vs 69%); this trend was seen at each time point. Overall, the proportion of children with high adherence was greater in pre-pubertal children than in pubertal children (girls: 80% vs 70%; boys: 79% vs 65%) and a higher proportion of pubertal girls had high adherence versus pubertal boys (70% vs 65%). Children with high adherence had the highest transmission rates per number of days with information available: 0.029 versus 0.018 (medium) and 0.019 (low).

We used a validated method to fill an unmet need in recognizing the factors that affect adherence to r-hGH. We showed that high adherence to treatment over time and engagement with the device was seen in patients using easypod connect; however, some patient groups are at particular risk of lower adherence and engagement.

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