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Journal Articles Cancer Discovery Year : 2022

The European MAPPYACTS Trial: Precision Medicine Program in Pediatric and Adolescent Patients with Recurrent Malignancies

1 IGR - Institut Gustave Roussy
2 Département de cancérologie de l'enfant et de l'adolescent [Gustave Roussy]
3 Institut Curie [Paris]
4 Department of Medical Oncology,
5 Département de médecine oncologique [Gustave Roussy]
6 U830 - Unité de génétique et biologie des cancers
7 Plateforme de Bioinformatique [Gustave Roussy]
8 ITIC - Immunologie anti-tumorale et immunothérapie des cancers
9 ICAN - Unité de Recherche sur les Maladies Cardiovasculaires, du Métabolisme et de la Nutrition = Research Unit on Cardiovascular and Metabolic Diseases
10 CESP - Centre de recherche en épidémiologie et santé des populations
11 SBE - Service de biostatistique et d'épidémiologie
12 CREDO - Centre de Recherche et de Documentation sur l'Océanie
13 U981 - Biomarqueurs prédictifs et nouvelles stratégies moléculaires en thérapeutique anticancéreuse
14 LabEx LERMIT - Laboratoire d'Excellence en Recherche sur le Médicament et l'Innovation Thérapeutique [Châtenay-Malabry]
15 Département de biologie et pathologie médicales [Gustave Roussy]
16 CHLS - Centre Hospitalier Lyon Sud [CHU - HCL]
17 PMNCO - Prédicteurs moléculaires et nouvelles cibles en oncologie
18 Centre Léon Bérard [Lyon]
19 IHOPe - Institut d'hématologie et d'oncologie pédiatrique [CHU - HCL]
20 Biologie Cellulaire et Cancer
21 CHU Nantes - Centre hospitalier universitaire de Nantes
22 IRCCS - Istituto di Ricovero e Cura a Carattere Scientifico, Ospedale Casa Sollievo della Sofferenza [San Giovanni Rotondo]
23 U1018 (Équipe 2) - Oncostat
24 Direction de la recherche clinique [Gustave Roussy]
25 UMR 1015 - Immunologie des tumeurs et immunothérapie
26 UMR 8203 - Vectorologie et thérapeutiques anti-cancéreuses [Villejuif]
Tiphaine Adam De Beaumais
  • Function : Author
Anne C. Harttrampf
  • Function : Author
Aroa Soriano Fernandez
  • Function : Author
Imene Hezam
  • Function : Author
Cecile Chevassus
  • Function : Author
Nicolas André
  • Function : Author
Cormac Owens
  • Function : Author
Raquel Hladun-Alvaro
  • Function : Author

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 AcSe-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).
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hal-04085309 , version 1 (06-06-2023)

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Pablo Berlanga, Gaëlle Pierron, Ludovic Lacroix, Mathieu Chicard, Tiphaine Adam De Beaumais, et al.. The European MAPPYACTS Trial: Precision Medicine Program in Pediatric and Adolescent Patients with Recurrent Malignancies. Cancer Discovery, 2022, 12 (5), pp.1266-1281. ⟨10.1158/2159-8290.CD-21-1136⟩. ⟨hal-04085309⟩
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