Blood-Derived Liquid Biopsies Using Foundation One® Liquid CDx for Children and Adolescents with High-Risk Malignancies: A Monocentric Experience
by
, ,
Fanny Cahn
1, 
Gabriel Revon-Riviere
1,2,
Victoria Min
1,
Angélique Rome
1,
Pauline Filaine
1,2,
Annick Pelletier
2,
Sylvie Abed
1,
Jean-Claude Gentet
1,
Arnauld Verschuur
1 and
Nicolas André
1,2,3,* 1
Department of Pediatric Oncology, La Timone University Hospital of Marseille, Assistance Publique-Hôpitaux de Marseille, 13005 Marseille, France
2
Centre d’essais Précoces en Cancérologie de Marseille (CEPCM), CLIPP2, Assistance Publique-Hôpitaux de Marseille, 13005 Marseille, France
3
SMARTc Unit, CRCM Inserm 1068, CNRS UMR 7258, Aix-Marseille University, 13005 Marseille, France
*
Author to whom correspondence should be addressed.
Cancers 2022, 14(11), 2774; https://doi.org/10.3390/cancers14112774
Submission received: 5 April 2022
/
Revised: 15 May 2022
/
Accepted: 22 May 2022
/
Published: 2 June 2022
(This article belongs to the Special Issue Targeted Therapies for Pediatric Solid Tumors)
Simple Summary
Precision oncology requires tumor molecular profiling to identify actionable targets. Blood-derived liquid biopsy (LB) is a potential alternative that is not yet documented in real-world settings, especially in pediatric oncology. Analyzing, retrospectively, the use of LB in children with refractory relapsing diseases, we were able to show that this is a feasible alternative to tissue biopsy, resulting in successful analysis in a subset of patients.
Abstract
Precision oncology requires tumor molecular profiling to identify actionable targets. Tumor biopsies are considered as the gold standard, but their indications are limited by the burden of procedures in children. Blood-derived liquid biopsy (LB) is a potential alternative that is not yet documented in real-world settings, especially in pediatric oncology. We performed a retrospective analysis of children and teenagers with a relapsing or refractory disease, upon whom LB was performed using the Foundation One® liquid CDx from 1 January 2020 to 31 December 2021 in a single center. Forty-five patients (27 boys) were included, with a median age of 9 years of age (range: 1.5–17 years old). Underlying malignancies were neuroblastoma (12 patients), bone sarcoma (12), soft tissue sarcoma (9), brain tumors (7), and miscellaneous tumors (5). Forty-three patients had metastatic disease. Six patients had more than one biopsy because of a failure in first LB. Median time to obtain results was 13 days. Overall, analysis was successful for 33/45 patients. Eight patients did not present any molecular abnormalities. Molecular alterations were identified in 25 samples with a mean of 2.1 alterations per sample. The most common alterations concerned TP53 (7 pts), EWS-FLI1 (5), ALK (3), MYC (3), and CREBBP (2). TMB was low in all cases. Six patients received treatment based on the results from LB analysis and all were treated off-trial. Three additional patients were included in early phase clinical trials. Mean duration of treatment was 85 days, with one patient with stable disease after eight months. Molecular profiling using Foundation One® Liquid CDx was feasible in pediatric patients with high-risk solid tumors and lead to identification of targetable mutations in a subset of patients.
