Evidence map›Paper›PMID 42543500›Full record

ArticleClinical endocrinology2026

Effectiveness of Recombinant Human Growth Hormone Therapy in Small-for-Gestational-Age Children With Short Stature: A Stratified Analysis Based on Genetic Variant Status.

Sanghee Park, Yena Lee, Hwal Rim Jeong, Eun Young Kim, Eu-Seon Noh, Hye Young Jin, Eun Byul Kwon, Hye Jin Lee, Young-Jun Seo, Young Suk Shim and 6 more

Abstract read
In one paragraph

Article in Clinical endocrinology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from 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.

2 · The registry

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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

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0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

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5 · Who and what money

Authors and funding

16 authors.

Sanghee ParkDepartment of Pediatrics, Hallym University Kangnam Sacred Heart Hospital, Seoul, Republic of Korea.
Yena LeeDepartment of Pediatrics, Hallym University Sacred Heart Hospital, Anyang, Republic of Korea.
Hwal Rim JeongDepartment of Pediatrics, College of Medicine, Soonchunhyang University, Cheonan, Republic of Korea.
Eun Young KimDepartment of Pediatrics, Chosun University Hospital, Gwangju, Republic of Korea.
Eu-Seon NohDepartment of Pediatrics, Kangdong Sacred Heart Hospital, Seoul, Republic of Korea.
Hye Young JinDepartment of Pediatrics, Kangdong Sacred Heart Hospital, Seoul, Republic of Korea.
Eun Byul KwonThe Key Growth Clinic, Seongnam, Gyeonggi-do, Republic of Korea.
Hye Jin LeeDepartment of Pediatrics, Hallym University Kangnam Sacred Heart Hospital, Seoul, Republic of Korea.
Young-Jun SeoDepartment of Pediatrics, Hallym University Chuncheon Sacred Heart Hospital, Chuncheon, Republic of Korea.
Young Suk ShimDepartment of Pediatrics, Ajou University Hospital, Ajou University School of Medicine, Suwon, Gyeonggi-do, Republic of Korea.
Su Jin KimDepartment of Pediatrics, Inha University Hospital, Inha University College of Medicine, Incheon, Korea.
Ji-Eun LeeDepartment of Pediatrics, Inha University Hospital, Inha University College of Medicine, Incheon, Korea.
Nan Young KimHallym Institute of Translational Genomics and Bioinformatics, Hallym University Medical Center, Anyang, Republic of Korea.
Sangkyoon HongHallym Institute of Translational Genomics and Bioinformatics, Hallym University Medical Center, Anyang, Republic of Korea.
Min Jae KangDepartment of Pediatrics, Hallym University Sacred Heart Hospital, Anyang, Republic of Korea.ORCID https://orcid.org/0000-0003-3080-0941
Il Tae HwangDepartment of Pediatrics, Kangdong Sacred Heart Hospital, Seoul, Republic of Korea.ORCID https://orcid.org/0000-0002-3885-4322

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveThis study aimed to evaluate the impact of pathogenic genetic variants on growth outcomes following 3 years of recombinant human growth hormone (rhGH) therapy in children born small for gestational age with persistent short stature (SGA-SS).

designA retrospective cohort study. PATIENTS: One hundred and seventy-nine SGA-SS children who underwent clinical and genetic evaluation were classified into variant-positive (n = 30) and variant-negative groups (n = 149). MEASUREMENTS: Clinical characteristics and growth outcomes were assessed over 3 years of rhGH therapy.

resultsAt baseline, the variant-positive group had significantly lower height standard deviation score (SDS) (-2.83 vs. -2.28, p < 0.001) and higher rates of intellectual disability (36.7% vs. 7.4%, p < 0.001) and congenital anomalies (30.0% vs. 6.7%, p < 0.001). rhGH therapy promoted longitudinal growth in both groups (evaluated n = 142); after 3 years, the estimated marginal mean (EMM) of height SDS reached -1.30 ± 0.10 in the variant-positive group and -0.93 ± 0.04 in the variant-negative group. However, linear mixed model analysis revealed a progressive attenuation of incremental height SDS gain in the variant-positive group as the treatment progressed, with significant interaction estimates observed at Year 1 (Estimate -0.18), Year 2 (Estimate -0.31) and Year 3 (Estimate -0.36; all p < 0.05).

conclusionAs one of the first longitudinal analyses utilizing repeated-measures modelling in a genetically characterized patient population, this study demonstrates that while rhGH therapy effectively promoted linear growth in SGA-SS children, patients with a molecular diagnosis presented with lower baseline stature and an attenuated incremental growth response over time, highlighting the importance of genetic evaluation for personalized treatment strategies.

Indexed as

DwarfismGrowth DisordersHuman Growth HormoneInfant, Small for Gestational AgeBody HeightChildChild, PreschoolFemaleGenetic VariationHumansInfant, NewbornMaleRecombinant ProteinsRetrospective StudiesTreatment OutcomeHuman Growth HormoneRecombinant Proteinsgenetic variantsgrowth hormoneshort staturesmall for gestational age

Identifiers

PMID42543500
PMCPMC13629597

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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.