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ABSTRACT: Background
Quantifying the association between adherence and the growth response to growth hormone (GH) treatment is hampered by suboptimal methods of measuring adherence, confounders associated with the growth response, and restriction of the outcome parameters to yearly growth velocities.Aim
To investigate the effect of adherence on the two-year growth response to GH treatment in prepubertal children with idiopathic isolated growth hormone deficiency (GHD) participating in the easypod connect observational study (ECOS), a 5-year, Phase IV open-label study to continuously assess real-world adherence via the easypod electronic drug-delivery device.Patients and methods
Outcome measures were change in height standard deviation score (?HSDS), index of responsiveness (IoR), and parameters of two catch-up growth (CUG) curve functions (monomolecular growth curve and second degree polynomial) with adj-HSDS (HSDS minus Target height (TH) SDS) as dependent variable. Inclusion criteria were GHD, naïve to GH treatment, known TH, age <10y in girls and <12y in boys, ?3 measurements, HSDS <-2 at start, complete data on growth and adherence in the first and second year. Linear regression analyses were performed to test the association between adherence (continuous and high vs. low) and the outcome measures, also adjusted for potential clinical confounders (age at start, adj-HSDS at start, birth weight SDS, gestational age (<37 weeks vs ?37 weeks), GH dose, GH max (n = 58)). The formula of IoR already adjusts for confounders.Results
In total, 95 patients complied with the inclusion criteria. The strongest associations were found between high adherence in the second year (?91% as cut-off value) and IoR 2y (+0.62), and average adherence and high adherence (?78%) in the first two years and ?HSDS 0-2y (+0.11 SD per 1 injection/week, and +0.34 SD for high vs. low adherence).Conclusion
Suboptimal adherence negatively affected the growth response in the first two years of GH treatment.
SUBMITTER: van Dommelen P
PROVIDER: S-EPMC6200242 | biostudies-literature | 2018
REPOSITORIES: biostudies-literature
van Dommelen Paula P Koledova Ekaterina E Wit Jan M JM
PloS one 20181024 10
<h4>Background</h4>Quantifying the association between adherence and the growth response to growth hormone (GH) treatment is hampered by suboptimal methods of measuring adherence, confounders associated with the growth response, and restriction of the outcome parameters to yearly growth velocities.<h4>Aim</h4>To investigate the effect of adherence on the two-year growth response to GH treatment in prepubertal children with idiopathic isolated growth hormone deficiency (GHD) participating in the ...[more]