Aubrie Eaton1, Victor Wong2, Deborah Schiff2, Eric Anderson2, Hilda Ding2, Edmund V Capparelli3, Deb Determan1, Dennis John Kuo2. 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. Copyright. Pediatric Pharmacy Association. All rights reserved. For permissions, email: mhelms@pediatricpharmacy.org 2022.
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. Copyright. Pediatric Pharmacy Association. All rights reserved. For permissions, email: mhelms@pediatricpharmacy.org 2022.
Entities:
Keywords:
acute lymphoblastic leukemia; acute myeloid leukemia; drug screening assays, antitumor; genomic profiling; pediatric oncology; precision medicine
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