Evidence map›Paper›PMID 41505353›Full record

ArticleHormone research in paediatrics2026

Longitudinal Observation of Children with Achondroplasia: Findings from a Global Natural History Study (ACHieve).

Ciara McDonnell, Hanne Buciek Hove, Melita Irving, Klane K White, Cesar G Fontecha, Janet M Legare, Wolfgang Högler, Daniel G Hoernschemeyer, Dirk Schnabel, Sheila Unger and 14 more

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Article in Hormone research in paediatrics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–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. Review
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

24 authors.

Ciara McDonnellDiscipline of Paediatrics, Children's Health Ireland at Temple Street, Dublin, Ireland.
Hanne Buciek HoveDepartment of Clinical Genetics, Copenhagen University Hospital Rigshospitalet, Copenhagen, Denmark.
Melita IrvingGuy's and St Thomas' NHS Foundation Trust, Evelina Children's Hospital, London, UK.
Klane K WhiteDepartment of Pediatric Orthopedic Surgery, Children's Hospital Colorado, Aurora, Colorado, USA.
Cesar G FontechaOrthopaedics and Traumatology Service, Hospital Sant Joan de Déu, Barcelona, Spain.
Janet M LegareDepartment of Pediatrics, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin, USA.
Wolfgang HöglerDepartment of Pediatrics and Adolescent Medicine, Johannes Kepler University Linz, Linz, Austria.
Daniel G HoernschemeyerPediatric Orthopedics, University of Missouri Children's Hospital, Columbia, Missouri, USA.
Dirk SchnabelCenter for Chronically Sick Children, Charité - University Medicine Berlin, Berlin, Germany.
Sheila UngerDivision of Genetic Medicine, Lausanne University Hospital, Lausanne, Switzerland.
Carlos Alberto BacinoDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, Texas, USA.
Paul HofmanThe Liggins Institute, University of Auckland, Auckland, New Zealand.
Yongguo YuDepartment of Pediatric Endocrinology and Genetics, Shanghai Institute for Pediatric Research, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Huamei MaDepartment of Pediatrics, The First Affiliated Hospital of Sun Yat-Sen University, Guangzhou, China.
Chunxiu GongDepartment of Endocrine and Genetics and Metabolism, Beijing Children's Hospital, Capital Medical University, National Centre for Children's Health, Beijing, China.
Xiaoping LuoDepartment of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
T Andrew BurrowDepartment of Pediatrics, University of Arkansas for Medical Sciences College of Medicine, Little Rock, Arkansas, USA.
Geneviève BaujatService de Médecine Génomique des Maladies Rares Hôpital Universitaire Necker-Enfants Malades, Institut Imagine, Université Paris Cité, Paris, France.
Stefano MoraDepartment of Pediatrics, Laboratory of Pediatric Endocrinology, IRCCS Ospedale San Raffaele, Milan, Italy.
Melissa FiscalettiCHU Sainte-Justine Azrieli Research Centre, University of Montreal, Montreal, Québec, Canada.
Carol ZhaoAscendis Pharma Inc., Palo Alto, California, USA.
Michael A MakaraAscendis Pharma Inc., Palo Alto, California, USA.
Aimee D ShuAscendis Pharma Inc., Palo Alto, California, USA.
Ravi SavarirayanSkeletal Biology, Murdoch Children's Research Institute, Royal Children's Hospital, Parkville, Victoria, Australia, ravi.savarirayan@mcri.edu.au.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionThe ACHieve study assessed growth velocity, body proportionality, and clinical events in children with achondroplasia not receiving growth-promoting therapy.

methodsACHieve was a global, longitudinal, prospective, observational study. Children ≤8 years old with achondroplasia were enrolled and evaluated every 6 months for anthropometric parameters and clinical events.

resultsACHieve enrolled 259 children in 15 countries, including 83 from China. Median follow-up was 21 months; median age of diagnosis was approximately 52 weeks in China and 2 weeks elsewhere. Growth parameters were similar regardless of region. Mean annualized growth velocity was 9.3 cm/year for males and 10.4 cm/year for females at age 1 and decreased to 4.1 cm/year and 4.6 cm/year, respectively, at age 4. Upper-to-lower-body segment ratio was generally consistent across regions. Overall, 77.2% of participants experienced clinical events, 34.0% of which were considered related to achondroplasia. Two deaths occurred (one accident and one cardiac arrest of unknown origin).

conclusionACHieve was one of the largest longitudinal natural history studies of achondroplasia to date and included the largest prospective Chinese achondroplasia cohort. The results demonstrated common trajectories in growth parameters regardless of region, indicating the generalizability of findings.

Indexed as

AchondroplasiaAnnualized growth velocityLongitudinalNatural history studyObservationalProspective

Identifiers

PMID41505353
PMCPMC12904650

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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.