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

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 i...

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Main Authors: Paula van Dommelen, Ekaterina Koledova, Jan M Wit
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2018-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC6200242?pdf=render
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author Paula van Dommelen
Ekaterina Koledova
Jan M Wit
author_facet Paula van Dommelen
Ekaterina Koledova
Jan M Wit
author_sort Paula van Dommelen
collection DOAJ
description 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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spelling doaj.art-c080ce6958da4310a96bf4964e98257d2022-12-21T22:26:24ZengPublic Library of Science (PLoS)PLoS ONE1932-62032018-01-011310e020600910.1371/journal.pone.0206009Effect of adherence to growth hormone treatment on 0-2 year catch-up growth in children with growth hormone deficiency.Paula van DommelenEkaterina KoledovaJan M WitBACKGROUND: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.http://europepmc.org/articles/PMC6200242?pdf=render
spellingShingle Paula van Dommelen
Ekaterina Koledova
Jan M Wit
Effect of adherence to growth hormone treatment on 0-2 year catch-up growth in children with growth hormone deficiency.
PLoS ONE
title Effect of adherence to growth hormone treatment on 0-2 year catch-up growth in children with growth hormone deficiency.
title_full Effect of adherence to growth hormone treatment on 0-2 year catch-up growth in children with growth hormone deficiency.
title_fullStr Effect of adherence to growth hormone treatment on 0-2 year catch-up growth in children with growth hormone deficiency.
title_full_unstemmed Effect of adherence to growth hormone treatment on 0-2 year catch-up growth in children with growth hormone deficiency.
title_short Effect of adherence to growth hormone treatment on 0-2 year catch-up growth in children with growth hormone deficiency.
title_sort effect of adherence to growth hormone treatment on 0 2 year catch up growth in children with growth hormone deficiency
url http://europepmc.org/articles/PMC6200242?pdf=render
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AT ekaterinakoledova effectofadherencetogrowthhormonetreatmenton02yearcatchupgrowthinchildrenwithgrowthhormonedeficiency
AT janmwit effectofadherencetogrowthhormonetreatmenton02yearcatchupgrowthinchildrenwithgrowthhormonedeficiency