{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,2]],"date-time":"2026-10-02T13:46:17Z","timestamp":1790948777371,"version":"4.1.0"},"reference-count":42,"publisher":"MDPI AG","issue":"10","license":[{"start":{"date-parts":[[2023,10,11]],"date-time":"2023-10-11T00:00:00Z","timestamp":1696982400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Carnot Curie Cancer"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Current Oncology"],"abstract":"<jats:p>Uveal melanoma is the most common primary intraocular malignancy in adults. Up to 50% of UM patients develop metastatic disease, usually in the liver. When metastatic, the prognosis is poor, and few treatment options exist. Here, we investigated the feasibility of establishing patient-derived xenografts (PDXs) from a patient\u2019s tumor in order to screen for therapies that the patient could benefit from. Samples obtained from 29 primary tumors and liver metastases of uveal melanoma were grafted into SCID mice. PDX models were successfully established for 35% of primary patient tumors and 67% of liver metastases. The tumor take rate was proportional to the risk of metastases. PDXs showed the same morphology, the same GNAQ\/11, BAP1, and SF3B1 mutations, and the same chromosome 3 and 8q status as the corresponding patient samples. Six PDX models were challenged with two compounds for 4 weeks. We show that, for 31% of patients with high or intermediate risk of metastasis, the timing to obtain efficacy results on PDX models derived from their primary tumors was compatible with the selection of the therapy to treat the patient after relapse. PDXs could thus be a valid tool (\u201cavatar\u201d) to select the best personalized therapy for one third of patients that are most at risk of relapse.<\/jats:p>","DOI":"10.3390\/curroncol30100657","type":"journal-article","created":{"date-parts":[[2023,10,11]],"date-time":"2023-10-11T08:18:57Z","timestamp":1697012337000},"page":"9090-9103","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Patient Derived Xenografts (PDX) Models as an Avatar to Assess Personalized Therapy Options in Uveal Melanoma: A Feasibility Study"],"prefix":"10.3390","volume":"30","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-8879-7592","authenticated-orcid":false,"given":"Fariba","family":"Nemati","sequence":"first","affiliation":[{"name":"Laboratory of Preclinical Investigation, Translational Research Department, Institut Curie, PSL University Paris, 26 rue d\u2019Ulm, CEDEX 05, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8072-3633","authenticated-orcid":false,"given":"Leanne","family":"de Koning","sequence":"additional","affiliation":[{"name":"Translational Research Department, Institut Curie, PSL University Paris, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8317-5188","authenticated-orcid":false,"given":"David","family":"Gentien","sequence":"additional","affiliation":[{"name":"Genomics Platform, Translational Research Department, Institut Curie, PSL Research University, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Franck","family":"Assayag","sequence":"additional","affiliation":[{"name":"Laboratory of Preclinical Investigation, Translational Research Department, Institut Curie, PSL University Paris, 26 rue d\u2019Ulm, CEDEX 05, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emilie","family":"Henry","sequence":"additional","affiliation":[{"name":"Genomics Platform, Translational Research Department, Institut Curie, PSL Research University, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Khadija","family":"Ait Rais","sequence":"additional","affiliation":[{"name":"Department of Genetics, Institut Curie, PSL Research University, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gaelle","family":"Pierron","sequence":"additional","affiliation":[{"name":"Department of Genetics, Institut Curie, PSL Research University, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Odette","family":"Mariani","sequence":"additional","affiliation":[{"name":"Biological Resource Center, Department of Pathology, Institut Curie, PSL Research University, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mich\u00e8le","family":"Nijnikoff","sequence":"additional","affiliation":[{"name":"Biological Resource Center, Department of Pathology, Institut Curie, PSL Research University, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1731-5343","authenticated-orcid":false,"given":"Gabriel","family":"Champenois","sequence":"additional","affiliation":[{"name":"Department of Biopathology, Institut Curie, PSL Research University, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andr\u00e9","family":"Nicolas","sequence":"additional","affiliation":[{"name":"Department of Biopathology, Institut Curie, PSL Research University, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Didier","family":"Meseure","sequence":"additional","affiliation":[{"name":"Department of Biopathology, Institut Curie, PSL Research University, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sophie","family":"Gardrat","sequence":"additional","affiliation":[{"name":"Department of Biopathology, Institut Curie, PSL Research University, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1678-7410","authenticated-orcid":false,"given":"Nicolas","family":"Servant","sequence":"additional","affiliation":[{"name":"Institut Curie, INSERM U900, CBIO-Centre for Computational Biology, Mines Paris Tech, PSL-Research University, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8468-3424","authenticated-orcid":false,"given":"Philippe","family":"Hup\u00e9","sequence":"additional","affiliation":[{"name":"Institut Curie, INSERM U900, CBIO-Centre for Computational Biology, Mines Paris Tech, PSL-Research University, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1466-0950","authenticated-orcid":false,"given":"Maud","family":"Kamal","sequence":"additional","affiliation":[{"name":"Department of Drug Development and Innovation (D3i), Institut Curie, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9772-4686","authenticated-orcid":false,"given":"Christophe","family":"Le Tourneau","sequence":"additional","affiliation":[{"name":"Department of Drug Development and Innovation (D3i), Institut Curie, 75248 Paris, France"},{"name":"INSERM U900 Research Unit, Institut Curie, 92064 Saint-Cloud, France"},{"name":"Paris-Saclay University, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sophie","family":"Piperno-Neumann","sequence":"additional","affiliation":[{"name":"Department of Medical Oncology, Institut Curie, PSL Research University, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5443-0802","authenticated-orcid":false,"given":"Manuel","family":"Rodrigues","sequence":"additional","affiliation":[{"name":"Department of Medical Oncology, Institut Curie, PSL Research University, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sergio","family":"Roman-Roman","sequence":"additional","affiliation":[{"name":"Translational Research Department, Institut Curie, PSL University Paris, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Didier","family":"Decaudin","sequence":"additional","affiliation":[{"name":"Laboratory of Preclinical Investigation, Translational Research Department, Institut Curie, PSL University Paris, 26 rue d\u2019Ulm, CEDEX 05, 75248 Paris, France"},{"name":"Department of Medical Oncology, Institut Curie, PSL Research University, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pascale","family":"Mariani","sequence":"additional","affiliation":[{"name":"Department of Surgical Oncology, Institut Curie, PSL Research University, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nathalie","family":"Cassoux","sequence":"additional","affiliation":[{"name":"Department of Oncological Ophthalmology, Institut Curie, Universit\u00e9 Paris Cit\u00e9, 75248 Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2023,10,11]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"2113","DOI":"10.2147\/OPTH.S113623","article-title":"Trends in incidence, survival, and management of uveal melanoma: A population-based study of 7,516 patients from the Surveillance, Epidemiology, and End Results database (1973\u20132012)","volume":"10","author":"Mahendraraj","year":"2016","journal-title":"Clin. Ophthalmol."},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"4651","DOI":"10.1167\/iovs.03-0538","article-title":"Very Long-Term Prognosis of Patients with Malignant Uveal Melanoma","volume":"44","author":"Kujala","year":"2003","journal-title":"Investig. Ophthalmol. Vis. Sci."},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"38","DOI":"10.1136\/bjophthalmol-2016-309034","article-title":"Metastatic disease from uveal melanoma: Treatment options and future prospects","volume":"101","author":"Carvajal","year":"2017","journal-title":"Br. J. Ophthalmol."},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"1192","DOI":"10.1016\/j.ejso.2009.02.016","article-title":"Surgical management of liver metastases from uveal melanoma: 16 years\u2019 experience at the Institut Curie","volume":"35","author":"Mariani","year":"2009","journal-title":"Eur. J. Surg. Oncol."},{"key":"ref_5","doi-asserted-by":"crossref","first-page":"360","DOI":"10.1159\/000508382","article-title":"Gene Expression Profiling in Uveal Melanoma: Five-Year Prospective Outcomes and Meta-Analysis","volume":"6","author":"Aaberg","year":"2020","journal-title":"Ocul. Oncol. Pathol."},{"key":"ref_6","doi-asserted-by":"crossref","first-page":"769","DOI":"10.1136\/bjophthalmol-2013-303867","article-title":"Genome-wide profiling is a clinically relevant and affordable prognostic test in posterior uveal melanoma","volume":"98","author":"Cassoux","year":"2014","journal-title":"Br. J. Ophthalmol."},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"1370","DOI":"10.1093\/annonc\/mdz176","article-title":"Meta-Analysis in Metastatic Uveal Melanoma to Determine Progression-Free and Overall Survival Benchmarks: An International Rare Cancers Initiative (IRCI) Ocular Melanoma study","volume":"31","author":"Khoja","year":"2019","journal-title":"Ann. Oncol."},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"1881","DOI":"10.1016\/j.ophtha.2011.01.040","article-title":"Uveal melanoma: Trends in incidence, treatment, and survival","volume":"118","author":"Singh","year":"2011","journal-title":"Ophthalmology"},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1007\/BF01200719","article-title":"Response of a multidrug-resistant human small-cell lung cancer xenograft to chemotherapy","volume":"120","author":"Arvelo","year":"1993","journal-title":"J. Cancer Res. Clin. Oncol."},{"key":"ref_10","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.canlet.2013.10.010","article-title":"One mouse, one patient paradigm: New avatars of personalized cancer therapy","volume":"344","author":"Malaney","year":"2014","journal-title":"Cancer Lett."},{"key":"ref_11","first-page":"3","article-title":"Patient-derived xenograft mouse models: A high fidelity tool for individualized medicine","volume":"17","author":"Xu","year":"2019","journal-title":"Oncol. Lett."},{"key":"ref_12","doi-asserted-by":"crossref","unstructured":"Koga, Y., and Ochiai, A. (2019). Systematic Review of Patient-Derived Xenograft Models for Preclinical Studies of Anti-Cancer Drugs in Solid Tumors. Cells, 8.","DOI":"10.3390\/cells8050418"},{"key":"ref_13","doi-asserted-by":"crossref","first-page":"338","DOI":"10.1038\/nrclinonc.2012.61","article-title":"Patient-derived tumour xenografts as models for oncology drug development","volume":"9","author":"Tentler","year":"2012","journal-title":"Nat. Rev. Clin. Oncol."},{"key":"ref_14","doi-asserted-by":"crossref","first-page":"5884","DOI":"10.1158\/0008-5472.CAN-19-1166","article-title":"A Comprehensive PDX Gastric Cancer Collection Captures Cancer Cell-Intrinsic Transcriptional MSI Traits","volume":"79","author":"Corso","year":"2019","journal-title":"Cancer Res."},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"2595","DOI":"10.1093\/annonc\/mdx416","article-title":"Patient-derived xenografts effectively capture responses to oncology therapy in a heterogeneous cohort of patients with solid tumors","volume":"28","author":"Izumchenko","year":"2017","journal-title":"Ann. Oncol."},{"key":"ref_16","doi-asserted-by":"crossref","first-page":"2476","DOI":"10.1158\/1078-0432.CCR-13-3047","article-title":"Integrated next-generation sequencing and avatar mouse models for personalized cancer treatment","volume":"20","author":"Garralda","year":"2014","journal-title":"Clin. Cancer Res."},{"key":"ref_17","doi-asserted-by":"crossref","first-page":"998","DOI":"10.1158\/2159-8290.CD-14-0001","article-title":"Patient-derived xenograft models: An emerging platform for translational cancer research","volume":"4","author":"Hidalgo","year":"2014","journal-title":"Cancer Discov."},{"key":"ref_18","doi-asserted-by":"crossref","first-page":"508","DOI":"10.1158\/2159-8290.CD-11-0109","article-title":"A molecularly annotated platform of patient-derived xenografts (\u201cxenopatients\u201d) identifies HER2 as an effective therapeutic target in cetuximab-resistant colorectal cancer","volume":"1","author":"Bertotti","year":"2011","journal-title":"Cancer Discov."},{"key":"ref_19","doi-asserted-by":"crossref","unstructured":"Inoue, A., Deem, A.K., Kopetz, S., Heffernan, T.P., Draetta, G.F., and Carugo, A. (2019). Current and Future Horizons of Patient-Derived Xenograft Models in Colorectal Cancer Translational Research. 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Cancers, 15.","DOI":"10.3390\/cancers15112989"}],"container-title":["Current Oncology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/1718-7729\/30\/10\/657\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,10]],"date-time":"2025-10-10T21:04:54Z","timestamp":1760130294000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/1718-7729\/30\/10\/657"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,10,11]]},"references-count":42,"journal-issue":{"issue":"10","published-online":{"date-parts":[[2023,10]]}},"alternative-id":["curroncol30100657"],"URL":"https:\/\/doi.org\/10.3390\/curroncol30100657","relation":{},"ISSN":["1718-7729"],"issn-type":[{"value":"1718-7729","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,10,11]]}}}