Evidence map›Paper›PMID 41326671›Full record

ArticleEuropean journal of pediatrics2025

Growth hormone treatment outcomes in children with genetic isolated growth hormone deficiency.

Karine Aouchiche, Sarah Castets, Isabelle Oliver Petit, Anya Rothenbuler, Patricia Bretones, Natacha Bouhours-Nouet, Thomas Edouard, Francois Brezin, Elsa Boncompain, Catherine Fabre-Brue and 5 more

Abstract read
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In one paragraph

Article in European journal of pediatrics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

15 authors.

Karine AouchicheUMR 1251, MMG, INSERM, Reference Center for Pituitary Rare Diseases HYPO La Timone Children's Hospital, APHM, Aix Marseille Univ, 264 Rue Saint Pierre, Marseille, 13385, France. Karine.aouchiche@ap-hm.fr.
Sarah CastetsMultidisciplinary Pediatric Department, Reference Center for Pituitary Rare Diseases HYPO, La Timone Children's Hospital, APHM, Marseille, France.
Isabelle Oliver PetitEndocrine, Reference Center for Rare Diseases of Growth and Development, Bone Diseases and Genetics Unit, FIRENDO Network, Children's Hospital, Toulouse University Hospital, Toulouse, France.
Anya RothenbulerEndocrinology and Diabetes for Children, Reference Center for Pituitary Rare Diseases HYPO, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris AP-HP, Hôpitaux Universitaires Paris-Saclay, Le Kremlin-Bicêtre, France.
Patricia BretonesPediatric Endocrinology Unit, Reference Center for Pituitary Rare Diseases HYPO, Hospices Civils de Lyon (HCL)Hôpital Femme Mère Enfant Université Claude Bernard Lyon 1, Lyon, France.
Natacha Bouhours-NouetDepartment of Pediatric Endocrinology and Diabetology, Reference Center for Pituitary Rare Diseases HYPO, University Hospital of Angers, Angers, France.
Thomas EdouardEndocrine, Reference Center for Rare Diseases of Growth and Development, Bone Diseases and Genetics Unit, FIRENDO Network, Children's Hospital, Toulouse University Hospital, Toulouse, France.
Francois BrezinDepartment of Pediatric Endocrinology, University Hospital of Strasbourg, Strasbourg, France.
Elsa BoncompainPediatric Department, Hospital of Roanne, Roanne, France.
Catherine Fabre-BrueMultidisciplinary Pediatric Department, Reference Center for Pituitary Rare Diseases HYPO, La Timone Children's Hospital, APHM, Marseille, France.
Maylis LebeaultDepartment of Endocrinology, Diabetology and Nutrition, University Hospital of Angers, Angers Cedex 9, Angers, France.
Thierry BrueUMR 1251, Department of Endocrinology, MarMaRa Institute, Reference Center for Pituitary Rare Diseases HYPO, La Conception University Hospital, APHM, Aix Marseille Univ, INSERM, Marseille, MMG, France.
Cécile Thomas-TeinturierEndocrinology and Diabetes for Children, Reference Center for Pituitary Rare Diseases HYPO, Hôpital Bicêtre, Assistance Publique-Hôpitaux de Paris AP-HP, Hôpitaux Universitaires Paris-Saclay, Le Kremlin-Bicêtre, France.
Alexandru SaveanuUMR 1251, Laboratory of Molecular Biology GEnOPé, La Timone University Hospital, Reference Center for Pituitary Rare Diseases HYPO, Univ, APHM, Aix Marseille INSERM, Marseille, MMG, France.
Rachel ReynaudUMR 1251, MMG, INSERM, Reference Center for Pituitary Rare Diseases HYPO La Timone Children's Hospital, APHM, Aix Marseille Univ, 264 Rue Saint Pierre, Marseille, 13385, France.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The aim of this study is to analyze the clinical characteristics of isolated growth hormone deficiency (IGHD) patients with GH1, GHRHR, and GHSR variants and their response to growth hormone (GH) treatment. Growth characteristics were retrospectively analyzed from GH treatment initiation in the Genhypopit cohort with likely pathogenic or pathogenic variants in GH1, GHRHR, or GHSR. Twenty-one patients (GH1: n = 13, GHRHR: n = 4, GHSR: n = 4) were followed up for 8.9 years (0.4; 19.6). GHD was diagnosed earlier in patients with GH1 or GHRHR variants than in those with GHSR variants (mean age at diagnosis: 3.1 and 2.0 years vs. 6.9 years, respectively). Patients with a family history of IGHD tend to have less severe short stature at diagnosis (- 2.2 vs. - 3.2 SDS, p = 0.053). Total height gain was significantly higher in patients with GH1 and GHRHR variants (+ 3.4 and + 3.8 SDS) than in those with GHSR variants (+ 1.8 SDS; p = 0.047). Total height gain was also associated with more severe initial growth delay (p < 0.001), greater difference from target height (p = 0.003), and earlier treatment initiation (p = 0.006). Patients born small for gestational age (SGA) experienced a growth gain similar to patients born eutrophic without the need to increase GH doses. This height gain under GH treatment was higher than reported previously in patients with non-genetic IGHD.

conclusionIdentifying a genetic cause of IGHD, particularly those involving variants in GH1 and GHRHR, is associated with significant height gain under GH treatment, regardless of their SGA status. WHAT IS KNOWN: • Recombinant growth hormone reliably improves growth in children with GHD. WHAT IS NEW: • Children with genetic IGHD, have very favorable GH treatment responses, unaffected by SGA status. • There are genotype-specific differences in growth outcomes under treatment.

Indexed as

Dwarfism, PituitaryHuman Growth HormoneReceptors, NeuropeptideReceptors, Pituitary Hormone-Regulating HormoneBody HeightChildChild, PreschoolFemaleFollow-Up StudiesHumansInfantMaleRetrospective StudiesTreatment OutcomeHuman Growth HormoneReceptors, NeuropeptideReceptors, Pituitary Hormone-Regulating Hormonesomatotropin releasing hormone receptorGeneticGH treatmentGrowthHeight velocityIGHD

Identifiers

PMID41326671

What OpenQuestion holds

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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.