ESPE Abstracts (2019) 92 P1-221

ESPE2019 Poster Category 1 GH and IGFs (1) (11 abstracts)

Individual Patterns of Objectively Measured Adherence to Growth Hormone Treatment and its Effect on Growth in Prepubertal Children with Growth Hormone Deficiency

Paula van Dommelen 1 , Jan M Wit 2 & Ekaterina Koledova 3


1The Netherlands Organization for Applied Scientific Research TNO, Leiden, Netherlands. 2Leiden University Medical Center, Leiden, Netherlands. 3Merck Healthcare KGaA, Darmstadt, Germany


The easypod™ electromechanical injection in combination with the easypod Connect platform electronically records and transmits, in real time, accurate, objective records of the date, time and dose injected for patients receiving Growth Hormone (GH) for growth disorders, limiting the risk of misreporting and allowing physicians to accurately monitor patient behavior.

The aim was to study individual patterns of adherence from start treatment up to 24 months and their effect on growth in prepubertal children with growth hormone deficiency (GHD). Data on adherence (=100% x number of injections taken/number of injections prescribed) and growth uploaded to the easypod Connect platform or collected by the Easypod Connect Observational Study (ECOS), a 5-year, Phase IV open-label study, were included. Inclusion criteria for adherence and growth were age before onset of puberty (<10 yrs in girls and <12 in boys). Additional inclusion criteria for growth were GHD and naïve to r-hGH treatment (ECOS study). Latent class mixed models with adherence as outcome and time after start of treatment (linear, centered quadratic) were constructed. This statistical method groups the patients into classes based on their individual adherence pattern over time. Descriptive statistics for the adherence classes were provided in categories (low ≤56%, medium >56 – <85%, high ≥85%). Linear regression analyses were performed to compare change of height standard deviation score (HSDS) between adherence classes from start of treatment up to 6 to 24 months.

Adherence data were available for 6588, 3951, 2888, and 2068 patients and height data for 251, 223, 179, and 147 patients (GHD and naïve for GH treatment) between 0-6 months, 6-12 months, 12-18 months, and 18-24 months, respectively. The latent class mixed models constructed three classes. Class 1 (n=342) had on average a medium adherence between 0-6 months, a low adherence between 6-18 months and a medium adherence between 18-24 months. Class 2 (n=5968) maintained a high adherence. Class 3 (n=278) had on average a low adherence between 0-6 months, and adherence consistently increased until 18-24 months till a medium adherence. Significant differences in change in HSDS between 0-18 months (change HSDS= -0.52, P=0.002) and 0-24 months (change HSDS= -0.45, P=0.03) were found in class 1 compared to class 2. No other significant differences were found.

Three classes of individual patterns of adherence to growth hormone treatment over time were constructed. There was a significant effect of adherence on growth in prepubertal children with GHD.

Volume 92

58th Annual ESPE

Vienna, Austria
19 Sep 2019 - 21 Sep 2019

European Society for Paediatric Endocrinology 

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