Marques da Costa, Maria EugéniaZaidi, SakinaScoazec, Jean YvesDroit, RobinLim, Wan ChingMarchais, AntoninSalmon, JeromeCherkaoui, SarahMorscher, Raphael J.Laurent, AnouchkaMalinge, SébastienMercher, ThomasTabone Eglinger, SéverineGoddard, IsabellePflumio, FrancoiseCalvo, JulienRedini, FrancoiseEntz-Werlé, NatachaSoriano, AroaVillanueva, AlbertoCairo, StefanoChastagner, PascalMoro, MassimoOwens, CormacCasanova, MichelaHladun Alvaro, RaquelBerlanga, PabloDaudigeos Dubus, EstelleDessen, PhilippeZitvogel, LaurenceLacroix, LudovicPierron, GaelleDelattre, OlivierSchleiermacher, GudrunSurdez, DidierGeoerger, Birgit2023-12-112023-12-112023-09-182399-3642https://hdl.handle.net/2445/204382Pediatric patients with recurrent and refractory cancers are in most need for new treatments. This study developed patient-derived-xenograft (PDX) models within the European MAPPYACTS cancer precision medicine trial (NCT02613962). To date, 131 PDX models were established following heterotopical and/or orthotopical implantation in immunocompromised mice: 76 sarcomas, 25 other solid tumors, 12 central nervous system tumors, 15 acute leukemias, and 3 lymphomas. PDX establishment rate was 43%. Histology, whole exome and RNA sequencing revealed a high concordance with the primary patient's tumor profile, human leukocyte-antigen characteristics and specific metabolic pathway signatures. A detailed patient molecular characterization, including specific mutations prioritized in the clinical molecular tumor boards are provided. Ninety models were shared with the IMI2 ITCC Pediatric Preclinical Proof-of-concept Platform (IMI2 ITCC-P4) for further exploitation. This PDX biobank of unique recurrent childhood cancers provides an essential support for basic and translational research and treatments development in advanced pediatric malignancies.15 p.application/pdfengcc by (c) Marques da Costa, Maria Eugénia et al., 2023http://creativecommons.org/licenses/by/3.0/es/CàncerPediatriaCancerPediatricsinfo:eu-repo/semantics/article2023-10-25info:eu-repo/semantics/openAccess37723198