Journal
NATURE MEDICINE
Volume 26, Issue 11, Pages 1742-+Publisher
NATURE PORTFOLIO
DOI: 10.1038/s41591-020-1072-4
Keywords
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Funding
- Australian Government
- Australian Federal Government Department of Health
- New South Wales State Government
- Australian Cancer Research Foundation
- Kids Cancer Alliance, Cancer Therapeutics Cooperative Research Centre
- Steven Walter Children's Cancer Foundation
- Hyundai Help 4 Kids Foundation
- Lions International
- ALCCRF
- University of New South Wales
- Australian Genomics Health Alliance
- New South Wales Ministry of Health
- Medical Research Future Fund
- Australian Brain Cancer Mission
- Minderoo Foundation's Collaborate Against Cancer Initiative
- Zero Childhood Cancer Capacity Campaign
- Children's Cancer Institute
- Sydney Children's Hospital Foundation
- National Health and Medical Research Council of Australia [APP1059804, APP1157871]
- Cancer Institute of New South Wales
- New South Wales Health
- Cancer Australia
- My Room [1165556]
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The Zero Childhood Cancer Program is a precision medicine program to benefit children with poor-outcome, rare, relapsed or refractory cancer. Using tumor and germline whole genome sequencing (WGS) and RNA sequencing (RNAseq) across 252 tumors from high-risk pediatric patients with cancer, we identified 968 reportable molecular aberrations (39.9% in WGS and RNAseq, 35.1% in WGS only and 25.0% in RNAseq only). Of these patients, 93.7% had at least one germline or somatic aberration, 71.4% had therapeutic targets and 5.2% had a change in diagnosis. WGS identified pathogenic cancer-predisposing variants in 16.2% of patients. In 76 central nervous system tumors, methylome analysis confirmed diagnosis in 71.1% of patients and contributed to a change of diagnosis in two patients (2.6%). To date, 43 patients have received a recommended therapy, 38 of whom could be evaluated, with 31% showing objective evidence of clinical benefit. Comprehensive molecular profiling resolved the molecular basis of virtually all high-risk cancers, leading to clinical benefit in some patients. The Zero Childhood Cancer pediatric precision medicine program informs treatment recommendations for children with high-risk cancers through comprehensive molecular profiling
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