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

耐火材料(行星科学) 医学 神经母细胞瘤 临床试验 癌症 肿瘤科 人口 内科学 生物 环境卫生 物理 天体生物学 遗传学 细胞培养
作者
Jordane Chaix,Gudrun Schleiermacher,Nadège Corradini,Nicolás André,Estelle Thébaud,Marion Gambart,Anne Sophie Defachelles,Natacha Entz‐Werlé,Pascal Chastagner,Émilie De Carli,Stéphane Ducassou,Judith Landman‐Parker,Tiphaine Adam de Beaumais,Alicia Larive,Stefan Michiels,Gilles Vassal,Dominique Valteau‐Couanet,Birgit Geoerger,Pablo Berlanga
出处
期刊:European Journal of Cancer [Elsevier BV]
卷期号:201: 113923-113923 被引量:1
标识
DOI:10.1016/j.ejca.2024.113923
摘要

Abstract

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
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