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Journal Articles JCO precision oncology Year : 2020

Tumor Molecular Profiling: Pediatric Results of the ProfiLER Study


PURPOSE The Program to Establish the Genetic and Immunologic Profile of Patient's Tumor for All Types of Advanced Cancer study (ClinicalTrials.gov identifier: NCT01774409 ) analyzed the genome of refractory cancers to identify a potential molecular-based recommended therapy (MBRT). The objectives of the pediatric substudy were to describe the incidence of genomic mutations, the MBRT, and the treatments undertaken with a molecular-targeted agent in a pediatric cohort. METHODS The tumor genome was analyzed within a 69-gene next-generation sequencing panel and an array comparative genomic hybridization assay. The results were evaluated by a multidisciplinary molecular board, and the targeted therapies were provided in the setting of a clinical trial or through compassionate use programs, when indicated. RESULTS Between November 2013 and June 2017, 50 patients younger than 19 years who were treated for a high-risk or relapsing tumor were included. Sarcomas (n = 24; 47%), CNS tumors (n = 14; 29%), and neuroblastomas (n = 5; 10%) were the most frequent tumor subtypes. Seven patients (14%) were excluded because no DNA could be recovered. Among the 43 remaining patients, 10 exhibited at least one targetable genomic alteration. Ultimately, four patients (8%) were treated with the recommended targeted therapy. CONCLUSION The results of this study confirm treatment with a targeted therapy for pediatric patients with cancer is still limited at present, as also is reported for adults.

Dates and versions

hal-03851844 , version 1 (14-11-2022)



Sarah Benezech, Pierre Saintigny, Valery Attignon, Daniel Pissaloux, Sandrine Paindavoine, et al.. Tumor Molecular Profiling: Pediatric Results of the ProfiLER Study. JCO precision oncology, 2020, 4, pp.785-795. ⟨10.1200/PO.20.00023⟩. ⟨hal-03851844⟩
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