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The European MAPPYACTS Trial: Precision Medicine Program in Pediatric and Adolescent Patients with Recurrent Malignancies
- Source :
- Scientia, Cancer Discovery, Cancer Discovery, 2022, 12 (5), pp.1266-1281. ⟨10.1158/2159-8290.CD-21-1136⟩
- Publication Year :
- 2022
- Publisher :
- American Association for Cancer Research (AACR), 2022.
-
Abstract
- Abstract MAPPYACTS (NCT02613962) is an international prospective precision medicine trial aiming to define tumor molecular profiles in pediatric patients with recurrent/refractory malignancies in order to suggest the most adapted salvage treatment. From February 2016 to July 2020, 787 patients were included in France, Italy, Ireland, and Spain. At least one genetic alteration leading to a targeted treatment suggestion was identified in 436 patients (69%) with successful sequencing; 10% of these alterations were considered “ready for routine use.” Of 356 patients with follow-up beyond 12 months, 107 (30%) received one or more matched targeted therapies—56% of them within early clinical trials—mainly in the AcSé-ESMART platform trial (NCT02813135). Overall, matched treatment resulted in a 17% objective response rate, and of those patients with ready for routine use alterations, it was 38%. In patients with extracerebral tumors, 76% of actionable alterations detected in tumor tissue were also identified in circulating cell-free DNA (cfDNA). Significance: MAPPYACTS underlines the feasibility of molecular profiling at cancer recurrence in children on a multicenter, international level and demonstrates benefit for patients with selected key drivers. The use of cfDNA deserves validation in prospective studies. Our study highlights the need for innovative therapeutic proof-of-concept trials that address the underlying cancer complexity. This article is highlighted in the In This Issue feature, p. 1171
- Subjects :
- neoplasias::procesos neoplásicos::recurrencia neoplásica local [ENFERMEDADES]
Pediatria
Adolescent
[SDV]Life Sciences [q-bio]
Carcinoma
High-Throughput Nucleotide Sequencing
Neoplasms [DISEASES]
neoplasias [ENFERMEDADES]
Càncer - Recaiguda
terapéutica::medicina de precisión [TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS]
Oncology
Mutation
Biomarkers, Tumor
Neoplasms::Neoplastic Processes::Neoplasm Recurrence, Local [DISEASES]
Humans
Prospective Studies
Medicina personalitzada
Neoplasm Recurrence, Local
Precision Medicine
Child
Therapeutics::Precision Medicine [ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT]
Cell-Free Nucleic Acids
Subjects
Details
- ISSN :
- 21598290 and 21598274
- Volume :
- 12
- Database :
- OpenAIRE
- Journal :
- Cancer Discovery
- Accession number :
- edsair.doi.dedup.....0d26fb3ca924e90aff48f3cd6b93979e