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. 2018 Oct 24;13(10):e0206009.
doi: 10.1371/journal.pone.0206009. eCollection 2018.

Effect of adherence to growth hormone treatment on 0-2 year catch-up growth in children with growth hormone deficiency

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

Effect of adherence to growth hormone treatment on 0-2 year catch-up growth in children with growth hormone deficiency

Paula van Dommelen et al. PLoS One. .

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 (ΔHSDS), 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 ΔHSDS 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.

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Conflict of interest statement

The authors have read the journal's policy and the authors of this manuscript have the following competing interests: PvD and JMW have consultancy agreements with Merck; EK is an employee of Merck KGaA, Darmstadt, Germany.

Figures

Fig 1
Fig 1. Selection criteria within the ECOS study.
Fig 2
Fig 2. Adherence in the first two years after starting growth hormone therapy.
See this image and copyright information in PMC

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