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Matched Targeted Therapy for Pediatric Patients with Relapsed, Refractory or High-risk Leukemias: A Report from the LEAP Consortium.

Pikman, Yana and Tasian, Sarah K and Sulis, Maria Luisa and Stevenson, Kristen and Blonquist, Traci M and Apsel Winger, Beth and Cooper, Todd M and Pauly, Melinda and Maloney, Kelly W and Burke, Michael J and Brown, Patrick A and Gossai, Nathan and McNeer, Jennifer L and Shukla, Neerav N and Cole, Peter D and Kahn, Justine M and Chen, Jing and Barth, Matthew J and Magee, Jeffrey A and Gennarini, Lisa and Adhav, Asmani A and Clinton, Catherine M and Ocasio-Martinez, Nicole and Gotti, Giacomo and Li, Yuting and Lin, Shan and Imamovic, Alma and Tognon, Cristina E and Patel, Tasleema and Faust, Haley L and Contreras, Cristina F and Cremer, Anjali and Cortopassi, Wilian A and Garrido Ruiz, Diego and Jacobson, Matthew P and Dharia, Neekesh V and Su, Angela and Robichaud, Amanda L and Saur Conway, Amy and Tarlock, Katherine and Stieglitz, Elliot and Place, Andrew E and Puissant, Alexandre and Hunger, Stephen P and Kim, Annette S and Lindeman, Neal I and Gore, Lia and Janeway, Katherine A and Silverman, Lewis B and Tyner, Jeffrey W and Harris, Marian H and Loh, Mignon L and Stegmaier, Kimberly (2021) Matched Targeted Therapy for Pediatric Patients with Relapsed, Refractory or High-risk Leukemias: A Report from the LEAP Consortium. Cancer discovery. ISSN 2159-8290

Abstract

Despite a remarkable increase in the genomic profiling of cancer, integration of genomic discoveries into clinical care has lagged behind. We report the feasibility of rapid identification of targetable mutations in 153 pediatric patients with relapsed/refractory or high-risk leukemias enrolled on a prospective clinical trial conducted by the LEAP Consortium. Eighteen percent of patients had a high confidence, Tier 1 or 2, recommendation. We describe clinical responses in the 14% of patients with relapsed/refractory leukemia who received the matched targeted therapy. Further, in order to inform future targeted therapy for patients, we validated variants of uncertain significance (VUS), performed ex vivo drug sensitivity testing in patient leukemia samples, and identified new combinations of targeted therapies in cell lines and patient-derived xenograft models. These data and our collaborative approach should inform the design of future precision medicine trials.

Item Type: Article
Date Deposited: 03 Mar 2021 00:45
Last Modified: 03 Mar 2021 00:45
URI: https://oak.novartis.com/id/eprint/44444

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