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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, Tasian, Sarah K., Sulis, Maria Luisa, Stevenson, Kristen, Blonquist, Traci M., Apsel Winger, Beth, Cooper, Todd M., Pauly, Melinda, Maloney, Kelly W., Burke, Michael J., Brown, Patrick A., Gossai, Nathan, McNeer, Jennifer L., Shukla, Neerav N., Cole, Peter D., Kahn, Justine M., Chen, Jing, Barth, Matthew J., Magee, Jeffrey A., Gennarini, Lisa, Adhav, Asmani A., Clinton, Catherine M., Ocasio-Martinez, Nicole, Gotti, Giacomo, Li, Yuting, Lin, Shan, Imamovic, Alma, Tognon, Cristina E., Patel, Tasleema, Faust, Haley L., Contreras, Cristina F., Cremer, Anjali, Cortopassi, Wilian A., Garrido Ruiz, Diego, Jacobson, Matthew P., Dharia, Neekesh V., Su, Angela, Robichaud, Amanda L., Saur Conway, Amy, Tarlock, Katherine, Stieglitz, Elliot, Place, Andrew E., Puissant, Alexandre, Hunger, Stephen P., Kim, Annette S., Lindeman, Neal I., Gore, Lia, Janeway, Katherine A., Silverman, Lewis B., Tyner, Jeffrey W., Harris, Marian H., 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, 11 (6). pp. 1424-1439. ISSN 2159-8274


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, 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: 22 Jul 2021 00:45
Last Modified: 22 Jul 2021 00:45


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