Division of Oncology and Children's Research Center, University Children's Hospital Zurich, University of Zurich, Zurich, Switzerland.
Cancer Discov. 2021 Jun;11(6):1322-1323. doi: 10.1158/2159-8290.CD-21-0258.
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..
皮克曼（Pikman）及其同事报告了白血病精准治疗联盟（LEAP）的一项多中心前瞻性临床试验结果，该联盟结合了耐药性儿童白血病中可靶向病变的鉴定，根据基因组病变和药物靶标证据进行分层，验证匹配的小分子靶向药物，并对个体患者进行治疗。该研究表明了基因组信息对疾病分类、治疗指导和转化研究的影响，但也说明了针对日益多样化和更小亚组患者进行目标预测和试验设计的挑战。
