Evidence map›Paper›PMID 39084206›Full record

ArticleHormone research in paediatrics2025

Accelerated Linear Growth during Erdafitinib Treatment: An FGFR-Related, but Growth Factor and Sex Steroid-Independent Mechanism?

Gabriele Hartmann, Adalbert Raimann, Natalia Stepien, Amedeo A Azizi, Gabriele Hartmann, Johannes Gojo

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Article in Hormone research in paediatrics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

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1citing papers in PubMed
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3 · Its place in the literature

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1 citing paper in PubMed.

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4 · The record

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5 · Who and what money

Authors and funding

6 authors.

Gabriele Hartmann
Adalbert RaimannDivision of Pediatric Pulmonology, Allergology and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University Vienna, Vienna, Austria.
Natalia StepienDepartment of Pediatrics and Adolescent Medicine, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, Vienna, Austria, natalia.stepien@meduniwien.ac.at.
Amedeo A AziziDepartment of Pediatrics and Adolescent Medicine, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, Vienna, Austria.
Gabriele HartmannDivision of Pediatric Pulmonology, Allergology and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University Vienna, Vienna, Austria.
Johannes GojoDepartment of Pediatrics and Adolescent Medicine, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, Vienna, Austria.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionGrowth acceleration during postnatal growth only occurs during puberty as a physiological event and during catch-up growth mediated by growth-promoting therapies in growth disorders. Here we report on novel observations of skeletal symptoms during treatment with erdafitinib, a tyrosine kinase inhibitor (TKI) prescribed on the basis of a compassionate-use program.

methodsAnalysis of anthropometric, biochemical, clinical, and radiographic data of patients with CNS tumors who revealed an unanticipated growth spurt with initiation of therapy with erdafitinib was performed retrospectively.

resultsLinear growth acceleration was independent of sex steroids and IGF1 levels, which is especially remarkable in the context of heavily pretreated pediatric neuro-oncology patients with severe growth impairment before initiation of therapy. Growth acceleration was accompanied by a distinct widening of the growth plate and enhanced metaphyseal mineralization shortly after the start of TKI therapy.

conclusionsWhile targeted therapies including TKIs have become an essential part of adult cancer treatment, applications in children are still limited. Off-target effects specific to the pediatric population have been observed in various organ systems; however, knowledge about the effect of TKIs on the growing skeleton is scarce. Treatment with erdafitinib inhibits FGFR3-mediated effects and thus represents a very logical hypothetical framework of growth factor and sex steroid-independent growth acceleration.

Indexed as

Insulin-Like Growth Factor IProtein Kinase InhibitorsPyrazolesQuinoxalinesReceptor, Fibroblast Growth Factor, Type 3AdolescentChildChild, PreschoolFemaleHumansMaleRetrospective StudieserdafitinibFGFR3 protein, humanIGF1 protein, humanInsulin-Like Growth Factor IProtein Kinase InhibitorsPyrazolesQuinoxalinesReceptor, Fibroblast Growth Factor, Type 3ErdafitinibFGFR inhibitionGrowth plateGrowth promotionLinear growthPediatric CNS tumors

Identifiers

PMID39084206
PMCPMC12668719

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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.