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The study developed patient-derived-xenograft (PDX) models within the European MAPPYACTS precision medicine trial (NCT02613962), establishing 131 models using heterotopic and/or orthotopic implantation in immunocompromised mice. Sequencing and histology showed high concordance with patients’ tumor profiles, HLA features, and metabolic pathway signatures, including prioritized actionable mutations. A subset was shared with the IMI2 ITCC Pediatric P4 platform to enable translational research and therapy development.",{"@graph":69,"@context":123},[70,84,106],{"@type":71,"itemListElement":72},"BreadcrumbList",[73,77,79,82],{"item":74,"name":75,"@type":76,"position":8},"https://docshare.wps.com","Home","ListItem",{"item":78,"name":9,"@type":76,"position":14},"https://docshare.wps.com/document/",{"item":80,"name":35,"@type":76,"position":81},"https://docshare.wps.com/document/healthcare/",3,{"item":83,"name":65,"@type":76,"position":19},"https://docshare.wps.com/document/a-biobank-of-pediatric-patient-derived-xenograft-models-in-cancer-precision-medicine-trial-mappyacts-for-relapsed-and-refractory-tumors/381644/",{"url":83,"name":65,"@type":85,"image":86,"author":91,"headline":65,"publisher":94,"fileFormat":97,"inLanguage":63,"description":67,"dateModified":98,"datePublished":99,"encodingFormat":97,"isAccessibleForFree":100,"interactionStatistic":101},"DigitalDocument",{"url":87,"@type":88,"width":89,"height":90},"https://docshare.wps.com/thumbnails/a-biobank-of-pediatric-patient-derived-xenograft-models-in-cancer-precision-medicine-trial-mappyacts-for-relapsed-and-refractory-tumors/381644.png","ImageObject",300,407,{"name":92,"@type":93},"Terk","Person",{"url":74,"name":95,"@type":96},"DocShare","Organization","application/pdf","2026-10-31","2026-09-24",true,{"@type":102,"interactionType":103,"userInteractionCount":105},"InteractionCounter",{"@type":104},"ViewAction",12,{"@type":107,"mainEntity":108},"FAQPage",[109,115,119],{"name":110,"@type":111,"acceptedAnswer":112},"What is the purpose of the MAPPYACTS PDX biobank for pediatric cancers?","Question",{"text":113,"@type":114},"It provides patient-derived xenograft models with deep phenotype characterization to support basic and translational research and to accelerate treatment development for advanced pediatric malignancies at the relapsed or refractory stage.","Answer",{"name":116,"@type":111,"acceptedAnswer":117},"How many PDX models were established and how were they generated?",{"text":118,"@type":114},"A total of 131 PDX models were established following heterotopic and/or orthotopic implantation in immunocompromised mice.",{"name":120,"@type":111,"acceptedAnswer":121},"What evidence shows that the PDX models match the original patient tumors?",{"text":122,"@type":114},"Histology and whole exome/RNA sequencing demonstrated high concordance with the primary patient’s tumor profile, human leukocyte-antigen characteristics, and specific metabolic pathway signatures.","https://schema.org",{"og:url":83,"og:type":125,"og:title":65,"og:site_name":95,"og:description":67},"article",{"robots":127,"canonical":83},"index,follow",{"doc_id":129,"site_id":62},381644,1790496188,{"code":4,"msg":5,"data":132},{"doc_id":129,"user_id":133,"nickname":92,"user_avatar":134,"doc_module":4,"category_id":34,"category_name":35,"doc_title":65,"doc_description":67,"doc_content":135,"file_id":136,"file_url":137,"file_type":138,"file_size":139,"view_count":105,"is_deleted":4,"is_public":8,"is_downloadable":8,"audit_status":8,"page_count":140,"language":141,"language_code":63,"site_id":62,"html_lang":63,"table_of_contents":142,"faqs":143,"seo_title":144,"seo_description":67,"update_tm":145,"read_time":146},1099525198933,"https://ap-avatar.wpscdn.com/davatar_155a257f0dc6eb9ab79c44ca47cae57d","ARTICLE   \n [https://doi.org/10.1038/s42003-023-05320-0](https://doi.org/10.1038/s42003-023-05320-0)  OPEN  \nA biobank of pediatric patient-derived-xenograft models in cancer precision medicine trial MAPPYACTS for relapsed and refractory tumors  \nMaria Eugénia Marques Da Costa  1,2, Sakina Zaidi  3, Jean-Yves Scoazec  4, Robin Droit1,5, Wan Ching Lim1,6, Antonin Marchais1,2, Jerome Salmon1, Sarah Cherkaoui1,7, Raphael J. Morscher1,7, Anouchka Laurent8, Sébastien Malinge  8,9, Thomas Mercher8, Séverine Tabone-Eglinger  10, Isabelle Goddard11, Francoise Pflumio 12, Julien Calvo  12, Francoise Redini13, Natacha Entz-Werlé14, Aroa Soriano15, Alberto Villanueva  16, Stefano Cairo17, Pascal Chastagner18, Massimo Moro  19,  \nCormac Owens20, Michela Casanova19, Raquel Hladun-Alvaro15, Pablo Berlanga2, Estelle Daudigeos-Dubus  1, Philippe Dessen1,5, Laurence Zitvogel  1, Ludovic Lacroix4, Gaelle Pierron  21, Olivier Delattre  3,21,22, Gudrun Schleiermacher  3,22, Didier Surdez  3,23,24 & Birgit Geoerger  1,2,24✉  \nPediatric 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.  \n*A list of author affiliations appears at the end of the paper.  \nCOMMUNICATIONS BIOLOGY | (2023)6:949 | [https://doi.org/10.1038/s42003-023-05320-0 |www.nature.com/commsbio](https://doi.org/10.1038/s42003-023-05320-0 |www.nature.com/commsbio) 1  \nnternationally standardized multimodal treatments now achieve a 5-year overall survival above 85% in children, ado  \nrelireeascspegy,nrtensaeantlyarldyybehoutwalf neogenfadtcauumncltesorswreithtysccpeapasencceanurrdr1e.Tdntehsfiispritstfielgiuhnrieeghtrholyeawetotmvxeeinrct  \neither by primary resistance or relapse after initial response.“MoleculAr Profiling for Pediatric and Young Adult Cancer Treatment Stratification” (MAPPYACTS; [ClinicalTrials.gov](ClinicalTrials.gov)[ ](ClinicalTrials.gov)[identi](identi)fi[er: NCT02613962](er: NCT02613962)) [is an international](is an international), [multicenter](multicenter), [pro](pro)  \nspective cancer precision medicine trial with the main objective to match relapsed or refractory pediatric patients to treatment with targeted agents based on individual advanced molecular tumor profiles2. A central ancillary study of the trial was the establishment of relevant preclinical patient-derived xenograft (PDX) models with deep phenotype characterization provided here.  \nPDX models, directly xenotransplanted from the primary patient tumor, are now widely in use as high impact preclinical models. They have been shown to retain tumor heterogeneity, histological characteristics, genomic profiles, as well as response patterns to therapies3–12. Despite the limitations that come with every model system, they have advanced our understanding in large cohorts of adult3, 13 as well as pediatric and adolescent cancer types4,7, 14, 15. A small fraction of reports highlights genomic discordances, with large chromosomal rearrangements or gen","cbCairhryusJf6ce","https://ap.wps.com/l/cbCairhryusJf6ce","pdf",2922449,15,"English","# Introduction\n## PDX models in precision medicine\n## Rationale for relapsed/refractory matched models\n# Results\n## Patient characteristics and PDX establishment","[{\"question\":\"What is the purpose of the MAPPYACTS PDX biobank for pediatric cancers?\",\"answer\":\"It provides patient-derived xenograft models with deep phenotype characterization to support basic and translational research and to accelerate treatment development for advanced pediatric malignancies at the relapsed or refractory stage.\"},{\"question\":\"How many PDX models were established and how were they generated?\",\"answer\":\"A total of 131 PDX models were established following heterotopic and/or orthotopic implantation in immunocompromised mice.\"},{\"question\":\"What evidence shows that the PDX models match the original patient tumors?\",\"answer\":\"Histology and whole exome/RNA sequencing demonstrated high concordance with the primary patient’s tumor profile, human leukocyte-antigen characteristics, and specific metabolic pathway signatures.\"}]","A biobank of pediatric patient-derived-xenograft models in cancer precision medicine trial MAPPYACTS for relapsed and refractory tumors | PDF",1790246242,38]