Feasibility of functional precision medicine for guiding treatment of relapsed or refractory pediatric cancers

Children with rare, relapsed or refractory cancers often face limited treatment options, and few predictive biomarkers are available that can enable personalized treatment recommendations. The implementation of functional precision medicine (FPM), which combines genomic profiling with drug sensitivi...

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Veröffentlicht in:Nature medicine 2024-04, Vol.30 (4), p.990-1000
Hauptverfasser: Acanda De La Rocha, Arlet M., Berlow, Noah E., Fader, Maggie, Coats, Ebony R., Saghira, Cima, Espinal, Paula S., Galano, Jeanette, Khatib, Ziad, Abdella, Haneen, Maher, Ossama M., Vorontsova, Yana, Andrade-Feraud, Cristina M., Daccache, Aimee, Jacome, Alexa, Reis, Victoria, Holcomb, Baylee, Ghurani, Yasmin, Rimblas, Lilliam, Guilarte, Tomás R., Hu, Nan, Salyakina, Daria, Azzam, Diana J.
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Sprache:eng
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Zusammenfassung:Children with rare, relapsed or refractory cancers often face limited treatment options, and few predictive biomarkers are available that can enable personalized treatment recommendations. The implementation of functional precision medicine (FPM), which combines genomic profiling with drug sensitivity testing (DST) of patient-derived tumor cells, has potential to identify treatment options when standard-of-care is exhausted. The goal of this prospective observational study was to generate FPM data for pediatric patients with relapsed or refractory cancer. The primary objective was to determine the feasibility of returning FPM-based treatment recommendations in real time to the FPM tumor board (FPMTB) within a clinically actionable timeframe (
ISSN:1078-8956
1546-170X
1546-170X
DOI:10.1038/s41591-024-02848-4