Evidence map›Paper›PMID 40626687›Full record

Observational studyThe Journal of clinical endocrinology and metabolism2026

Near-Adult Height Outcomes in Patients Treated With rhIGF-1 for Severe Growth Failure: Real-World IGFD Registry Data.

Marta Ramon-Krauel, Michel Polak, Mohamad Maghnie, Joachim Woelfle, Caroline Sert, Valérie Perrot, Peter Bang

Registry-linked trialAbstract readMulticenter StudyObservational Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study in The Journal of clinical endocrinology and metabolism, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00903110 (Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency), which is not on this map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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.

NCT00903110 recruitingnot on this map

Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD).

Typeobservational_patient_registrySponsorEsteve Pharmaceuticals, S.A.Ran2008 to 2027Enrolled500ConditionsIGF1 DeficiencyArmsIncrelex®
3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Article
  2. Observational
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

7 authors.

Marta Ramon-KrauelHospital Sant Joan de Déu, Institut de Recerca Sant Joan de Déu, 08950 Barcelona, Spain.ORCID 0000-0003-3027-9105
Michel PolakDepartment of Pediatric Endocrinology, Gynaecology, and Diabetology, Assistance Publique-Hôpitaux de Paris, Hôpital Universitaire Necker-Enfants Malades, IMAGINE Institute, INSERM U1016, Université Paris Cité, 75015 Paris, France.ORCID 0000-0003-2639-352X
Mohamad MaghnieEndo-ERN European Reference Network on Rare Endocrine Conditions, 1105 AZ Amsterdam, The Netherlands.ORCID 0000-0002-7183-5238
Joachim WoelfleDepartment of Pediatrics and Adolescent Medicine, Friedrich-Alexander-University Erlangen-Nürnberg, 91054 Erlangen, Germany.ORCID 0000-0002-9132-6174
Caroline SertIpsen, 92100 Boulogne-Billancourt, France.
Valérie PerrotIpsen, 92100 Boulogne-Billancourt, France.
Peter BangDivision of Pediatrics, Department of Biomedical and Clinical Sciences, Faculty of Health Sciences, Linköping University, 581 83 Linköping, Sweden.

Funding

Ipsen
6 · The paper itself

Abstract

contextThe Global Increlex® Growth Forum Database (IGFD) Registry monitors real-world effectiveness and safety of recombinant human IGF-1 (rhIGF-1; Increlex® [mecasermin]) treatment in children and adolescents with severe growth failure due to severe primary IGF-I deficiency (SPIGFD).

objectiveTo report characteristics, effectiveness, and safety data from patients receiving rhIGF-1 treatment who achieved near-adult height (NAH), and determine factors that predict height gain to NAH.

methodsDescriptive analyses of patients included in the Global IGFD Registry (NCT00903110) who achieved NAH are reported for the overall population, treatment-naïve prepubertal (NPP) patients, and patients with Laron syndrome. Linear regression analyses of height gain to NAH are also reported.

resultsOne hundred and two patients enrolled in the Global IGFD Registry achieved NAH at data cut-off (April 20, 2023). Mean age at rhIGF-1 treatment initiation was 11.8 years; median treatment duration was 3.9 years. Mean (SD) height SD score (HtSDS) gain from rhIGF-1 initiation to NAH was 0.9 (1.1). In NPP patients, mean (SD) HtSDS gain was 1.4 (1.0). Almost half of NPP patients reached NAH within the normal range. Despite improved height in patients with Laron syndrome, only 10.5% reached NAH within the normal range; 3 patients with Laron syndrome were NPP. Treatment naivety was predictive of height gain in the overall NAH population. Safety data aligned with previous reports.

conclusionIn a real-world setting, despite patients with SPIGFD initiating rhIGF-1 treatment at a relatively advanced age, rhIGF-1 treatment resulted in improved NAH. The greatest improvements in height outcomes were observed in NPP patients.

trial registrationNCT00903110.

Indexed as

Body HeightGrowth DisordersInsulin-Like Growth Factor IAdolescentAdultChildChild, PreschoolFemaleHearing Loss, SensorineuralHumansMaleRecombinant ProteinsRegistriesSeverity of Illness IndexTreatment OutcomeYoung AdultIGF1 protein, humanInsulin-Like Growth Factor ImecaserminRecombinant Proteinsadult heightglobal IGFD registryrare growth disordersevere primary insulin-like growth factor-I deficiency

Identifiers

PMID40626687
PMCPMC12819849

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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.