Trial reportEuropean journal of endocrinology2013
A pharmacogenomic approach to the treatment of children with GH deficiency or Turner syndrome.
Trial report in European journal of endocrinology, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00699855 (Observational Long-term Follow-up of the Phase IV Open-label Trial of Predictive Markers in GHD and TS Pre-pubertal Children Treated With Saizen), which is not on this map. Cited by 19 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Observational Long-term Follow-up of the Phase IV Open-label Trial of Predictive Markers in GHD and TS Pre-pubertal Children Treated With Saizen
Who cites it
19 citing papers in PubMed, 1 synthesis or guideline pooled it, 33 citations in OpenAlex.
- [Turner syndrome and genetic polymorphism: a systematic review].Revista paulista de pediatria : orgao oficial da Sociedade de Pediatria de Sao PauloPooled it
- Gene Expression Signatures Predict First-Year Response to Somapacitan Treatment in Children With Growth Hormone Deficiency.The Journal of clinical endocrinology and metabolism · 2024Trial
- Gene expression signatures predict response to therapy with growth hormone.The pharmacogenomics journal · 2021Trial
- Transcriptomics and machine learning predict diagnosis and severity of growth hormone deficiency.JCI insight · 2018Trial
- GH deficiency status combined with GH receptor polymorphism affects response to GH in children.European journal of endocrinology · 2015Trial
- Robust prediction of response to growth hormone therapy by five PBMC-based transcripts.Genes & diseases · 2027Article
- Transcriptome remodelling and changes in growth and cardiometabolic phenotype result following Grb10a knockdown in the early life of the zebrafish.Cellular and molecular life sciences : CMLS · 2025Article
- Response of children with Turner syndrome with different types of karyotype abnormalities to growth hormone treatment.Annals of pediatric endocrinology & metabolism · 2024Article
- Transforming Growth Factor Beta Receptor 2 (TGFBR2) Promoter Region Polymorphisms May Be Involved in Mandibular Retrognathism.BioMed research international · 2022Article
- Karyotype Abnormalities in the X Chromosome Predict Response to the Growth Hormone Therapy in Turner Syndrome.Journal of clinical medicine · 2021Article
- Pharmacogenomics applied to recombinant human growth hormone responses in children with short stature.Reviews in endocrine & metabolic disorders · 2021Review
- A Genome-Wide Pharmacogenetic Study of Growth Hormone Responsiveness.The Journal of clinical endocrinology and metabolism · 2020Observational
- The in vitro functional analysis of single-nucleotide polymorphisms associated with growth hormone (GH) response in children with GH deficiency.The pharmacogenomics journal · 2019Article
- Assessment of the key regulatory genes and their Interologs for Turner Syndrome employing network approach.Scientific reports · 2018Article
- Baseline IGFBP - 3 as the Key Element to Predict Growth Response to Growth Hormone and IGF - 1 Therapy in Subjects with Non - GH Deficient Short Stature and IGF - 1 Deficiency.International journal of endocrinology and metabolism · 2018Article
- Growth Hormone Research Society perspective on biomarkers of GH action in children and adults.Endocrine connections · 2018Review
- Diagnostic and therapeutic considerations in Turner syndrome.Annals of pediatric endocrinology & metabolism · 2017Review
- Validating genetic markers of response to recombinant human growth hormone in children with growth hormone deficiency and Turner syndrome: the PREDICT validation study.European journal of endocrinology · 2016Article
- Effect of summer daylight exposure and genetic background on growth in growth hormone-deficient children.The pharmacogenomics journal · 2016Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
13 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveIndividual sensitivity to recombinant human GH (r-hGH) is variable. Identification of genetic factors contributing to this variability has potential use for individualization of treatment. The objective of this study was to identify genetic markers and gene expression profiles associated with growth response on r-hGH therapy in treatment-naïve, prepubertal children with GH deficiency (GHD) or Turner syndrome (TS).
designA prospective, multicenter, international, open-label pharmacogenomic study.
methodsThe associations of genotypes in 103 growth- and metabolism-related genes and baseline gene expression profiles with growth response to r-hGH (cm/year) over the first year were evaluated. Genotype associations were assessed with growth response as a continuous variable and as a categorical variable divided into quartiles.
resultsEleven genes in GHD and ten in TS, with two overlapping between conditions, were significantly associated with growth response either as a continuous variable (seven in GHD, two in TS) or as a categorical variable (four more in GHD, eight more in TS). For example, in GHD, GRB10 was associated with high response (≥ Q3; P=0.0012), while SOS2 was associated with low response (≤ Q1; P=0.006), while in TS, LHX4 was associated with high response (P=0.0003) and PTPN1 with low response (P=0.0037). Differences in expression were identified for one of the growth response-associated genes in GHD (AKT1) and for two in TS (KRAS and MYOD1).
conclusionsCarriage of specific growth-related genetic markers is associated with growth response in GHD and TS. These findings indicate that pharmacogenomics could have a role in individualized management of childhood growth disorders.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.