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Comment
. 2021 Jun;11(6):1322-1323.
doi: 10.1158/2159-8290.CD-21-0258.

A Hopeful Leap Forward by Multicentric Cooperation for Precision-Based Therapy for Very Resistant, Relapsed, or Refractory Childhood Leukemia

Beat C Bornhauser  1 , Jean-Pierre Bourquin  2
Affiliations
Free article
Comment

A Hopeful Leap Forward by Multicentric Cooperation for Precision-Based Therapy for Very Resistant, Relapsed, or Refractory Childhood Leukemia

Beat C Bornhauser et al. Cancer Discov. 2021 Jun.
Free article

Abstract

Pikman and colleagues report the results of a multicentric prospective clinical trial of the leukemia precision-based therapy (LEAP) consortium that combines identification of targetable lesions in drug-resistant childhood leukemia, tiered based on evidence for genomic lesions and drug target, validation of matching small-molecule targeted agents, and treatment of individual patients. The study demonstrates the impact of genomic information on disease classification, treatment guidance, and translational research, but also illustrates the challenges for target prediction and trial design for increasingly heterogeneous and smaller subgroups of patients.See related article by Pikman et al., p. 1424.

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Comment on

  • Matched Targeted Therapy for Pediatric Patients with Relapsed, Refractory, or High-Risk Leukemias: A Report from the LEAP Consortium.
    Pikman Y, Tasian SK, Sulis ML, Stevenson K, Blonquist TM, Apsel Winger B, Cooper TM, Pauly M, Maloney KW, Burke MJ, Brown PA, Gossai N, McNeer JL, Shukla NN, Cole PD, Kahn JM, Chen J, Barth MJ, Magee JA, Gennarini L, Adhav AA, Clinton CM, Ocasio-Martinez N, Gotti G, Li Y, Lin S, Imamovic A, Tognon CE, Patel T, Faust HL, Contreras CF, Cremer A, Cortopassi WA, Garrido Ruiz D, Jacobson MP, Dharia NV, Su A, Robichaud AL, Saur Conway A, Tarlock K, Stieglitz E, Place AE, Puissant A, Hunger SP, Kim AS, Lindeman NI, Gore L, Janeway KA, Silverman LB, Tyner JW, Harris MH, Loh ML, Stegmaier K. Pikman Y, et al. Cancer Discov. 2021 Jun;11(6):1424-1439. doi: 10.1158/2159-8290.CD-20-0564. Epub 2021 Feb 9. Cancer Discov. 2021. PMID: 33563661 Free PMC article. Clinical Trial.

References

    1. Pui C-H, Yang JJ, Hunger SP, Pieters R, Schrappe M, Biondi A, et al. Childhood acute lymphoblastic leukemia: progress through collaboration. J Clin Oncol. 2015;33:2938–48.
    1. Sidhom I, Shaaban K, Youssef SH, Ali N, Gohar S, Rashed WM, et al. Reduced-intensity therapy for pediatric lymphoblastic leukemia: impact of residual disease early in remission induction. Blood. 2021;137:20–8.
    1. Valent P, Orfao A, Kubicek S, Staber P, Haferlach T, Deininger M, et al. Precision medicine in hematology 2021: definitions, tools, perspectives, and open questions. Hemasphere. 2021;5:e536.
    1. Wong M, Mayoh C, Lau LMS, Khuong-Quang D-A, Pinese M, Kumar A, et al. Whole genome, transcriptome and methylome profiling enhances actionable target discovery in high-risk pediatric cancer. Nat Med. 2020;26:1742–53.
    1. Pikman Y, Tasian SK, Sulis ML, Stevenson K, Blonquist TM, Apsel Winger B, et al. Matched targeted therapy for pediatric patients with relapsed, refractory, or high-risk leukemias: A report from the LEAP consortium. Cancer Discov. 2021;11:1424–39.

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