Clinical trial inclusion in patients with relapsed/refractory neuroblastoma following the European Precision Cancer Medicine trial MAPPYACTS.

Autor: Chaix J; Department of Pediatric and Adolescent Oncology, Gustave Roussy Cancer Campus, Université Paris-Saclay, Villejuif, France., Schleiermacher G; INSERM U830, Laboratoire de Génétique et Biologie des Cancers, Research Center, PSL Research University, Institut Curie, Paris, France; SIREDO Oncology Center (Care, Innovation and Research for Children and AYA with Cancer), Institut Curie, PSL Research University, Paris, France., Corradini N; Department of Pediatric Oncology, Institut d'Hématologie et d'Oncologie Pédiatrique/Centre Léon Bérard, Lyon, France., André N; Department of Pediatric Hematology and Oncology, Hôpital de La Timone, AP-HM, Marseille, France; UMR Inserm 1068, CNRS UMR 7258, Aix Marseille Université U105, Marseille Cancer Research Center (CRCM), Marseille, France., Thebaud E; Department of Pediatric Oncology, Centre Hospitalier Universitaire, Nantes, France., Gambart M; Department of Pediatric Oncology, Centre Hospitalier Universitaire, Toulouse, France., Defachelles AS; Department of Pediatric Oncology, Centre Oscar Lambret, Lille, France., Entz-Werle N; Department of Pediatric Oncology, Hospices Civils de Strasbourg, Strasbourg, France., Chastagner P; Department of Pediatric Oncology, Centre Hospitalier Universitaire, Vandoeuvre les Nancy, France., De Carli É; Department of Pediatric Oncology, Centre Hospitalier Universitaire, Angers, France., Ducassou S; Department of Pediatric Oncology, Centre Hospitalier Universitaire, Bordeaux, France., Landman-Parker J; Sorbonne Université APHP, Hôpital A Trousseau Paris, Paris, France., Adam-de-Beaumais T; Clinical Research Direction, Gustave Roussy Cancer Campus, Université Paris-Saclay, Villejuif, France., Larive A; Biostatistics and Epidemiology Office, Gustave Roussy Cancer Campus, INSERM U1018, CESP, Université Paris-Saclay, Villejuif, France., Michiels S; Biostatistics and Epidemiology Office, Gustave Roussy Cancer Campus, INSERM U1018, CESP, Université Paris-Saclay, Villejuif, France., Vassal G; Clinical Research Direction, Gustave Roussy Cancer Campus, Université Paris-Saclay, Villejuif, France., Valteau-Couanet D; Department of Pediatric and Adolescent Oncology, Gustave Roussy Cancer Campus, Université Paris-Saclay, Villejuif, France., Geoerger B; Department of Pediatric and Adolescent Oncology, Gustave Roussy Cancer Campus, Université Paris-Saclay, Villejuif, France; INSERM U1015, Gustave Roussy Cancer Campus, Université Paris-Saclay, Villejuif, France., Berlanga P; Department of Pediatric and Adolescent Oncology, Gustave Roussy Cancer Campus, Université Paris-Saclay, Villejuif, France. Electronic address: Pablo.Berlanga@gustaveroussy.fr.
Jazyk: angličtina
Zdroj: European journal of cancer (Oxford, England : 1990) [Eur J Cancer] 2024 Apr; Vol. 201, pp. 113923. Date of Electronic Publication: 2024 Feb 15.
DOI: 10.1016/j.ejca.2024.113923
Abstrakt: Introduction: Despite poor survival for patients with relapsed or refractory neuroblastoma, only 10-16% of patients are reported to be included in early phase trials. This study aimed to explore the impact of molecular profiling within the prospective precision cancer medicine trial MAPPYACTS (NCT02613962) on subsequent early phase trial recruitment and treatment by matched targeted therapies in this population.
Methods and Materials: Clinical data from all French patients with relapsed/refractory neuroblastoma enrolled in MAPPYACTS were analyzed for subsequent matched/non-matched targeted treatment based on clinical tumor board (CMTB) recommendations.
Results: From 93 patients with neuroblastoma included in French centers, 78 (84%) underwent whole exome and RNA sequencing and were discussed in the CMTB. Higher rate of successful sequencing analysis was observed in patients with relapsed disease compared to those with refractory disease (p = 0.0002). Among the 50 patients that presented with a new disease relapse/progression after the CMTB recommendations, 35 patients (70%) had at least one actionable alteration identified on the tumor at the time of relapse. Eighteen patients (36%) were included in an early phase clinical trial, 11 of these with a matched agent, 7 with a non-matched treatment; 13 patients were included in the AcSé ESMART trial. Five patients (10%) received a matched targeted therapy outside a clinical trial.
Conclusion: Patients with neuroblastoma in the European MAPPYACTS trial were more likely to be included in early phase trials compared to previous reports. Early deep sequencing at first treatment failure, comprehensive therapeutic discussions in molecular tumor boards and innovative trials like AcSé -ESMART improve access to innovative therapies for patients with relapsed/refractory neuroblastoma.
Clinical Trial Registration: NCT02613962.
Competing Interests: Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.
(Copyright © 2024. Published by Elsevier Ltd.)
Databáze: MEDLINE
Externí odkaz: