Tumor Genomic Profiling and Ex Vivo Drug Sensitivity Testing for Pediatric Leukemia and Lymphoma Patients

Aubrie Eaton; Victor Wong; Deborah Schiff; Eric Anderson; Hilda Ding; Edmund V Capparelli; Deb Determan; Dennis John Kuo

doi:10.5863/1551-6776-27.2.123

Tumor Genomic Profiling and Ex Vivo Drug Sensitivity Testing for Pediatric Leukemia and Lymphoma Patients

J Pediatr Pharmacol Ther. 2022;27(2):123-131. doi: 10.5863/1551-6776-27.2.123. Epub 2022 Feb 9.

Authors

Aubrie Eaton¹, Victor Wong², Deborah Schiff², Eric Anderson², Hilda Ding², Edmund V Capparelli³, Deb Determan¹, Dennis John Kuo²

Affiliations

¹ Department of Pharmacy (AE, DD), Rady Children's Hospital, San Diego, San Diego, CA.
² Department of Pediatrics, Division of Hematology-Oncology (VW, DS, EA, HD, DJK), University of California San Diego, La Jolla, CA/Rady Children's Hospital, San Diego, San Diego, CA.
³ Department of Pediatrics and Skaggs School of Pharmacy and Pharmaceutical Sciences (EVC), University of California San Diego, La Jolla, CA.

Abstract

Objective: To describe the frequency of use of tumor genomic profiling and functional ex vivo drug sensitivity testing in pediatric patients with hematologic malignancies at our institution, and to determine how the results affected treatment selection.

Methods: A retrospective chart review was conducted to analyze the frequency of tumor genomic profiling and functional drug sensitivity screening in our institution in pediatric patients with hematologic malignancies and to ask if the results were used to direct treatment. A case series of patients for whom these testing recommendations resulted in therapeutic interventions is reported.

Results: Thirty-three patients underwent tumor genomic profiling assays, functional ex vivo testing, or both. Nineteen patients (58%) had genomic profiling assays performed alone, 3 (9%) had functional ex vivo testing performed alone, and 11 (33%) had both tests performed. Twenty-one (64%) patients had potentially actionable mutations detected by the genomic profiling assay. Seven (21%) patients received at least 1 chemotherapeutic agent in accordance with the tumor genomic profiling or functional ex vivo drug sensitivity testing results. Three (43%) of the 7 patients who were treated with testing directed therapy had a favorable treatment response (PR or CR) to treatments selected based upon results of genomic or functional ex vivo testing.

Conclusions: This retrospective case series demonstrates that precision medicine techniques such as genomic profiling and drug sensitivity testing can positively inform treatment selection in pediatric patients with relapsed or refractory leukemia and lymphoma.

Keywords: acute lymphoblastic leukemia; acute myeloid leukemia; drug screening assays, antitumor; genomic profiling; pediatric oncology; precision medicine.