Accelerated Linear Growth during Erdafitinib Treatment: An FGFR-Related, but Growth Factor and Sex Steroid-Independent Mechanism?
Authors' conclusion
While 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.
Study context
- Population
- Heavily pretreated pediatric neuro-oncology patients with CNS tumors and severe growth impairment before therapy, who showed an unanticipated growth spurt with initiation of erdafitinib
- Outcome
- Linear growth and skeletal changes during erdafitinib
Methodological detailscompassionate-use program
- Compassionate-use program
Main finding
As reported in the abstract
- Linear growth acceleration was independent of sex steroids and IGF-1 levels
- Growth acceleration accompanied by distinct widening of the growth plate and enhanced metaphyseal mineralization shortly after start of tyrosine kinase inhibitor (TKI) therapy
Observational data: associations, not effects.
Original publication
Accelerated Linear Growth during Erdafitinib Treatment: An FGFR-Related, but Growth Factor and Sex Steroid-Independent Mechanism? Horm Res Paediatr. Published 31 July 2024. doi:10.
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