Precision-guided treatment in high-risk pediatric cancers.
- Resource Type
- Academic Journal
- Authors
- Lau LMS; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.; Kids Cancer Centre, Sydney Children's Hospital, Sydney, New South Wales, Australia.; Khuong-Quang DA; Children's Cancer Centre, Royal Children's Hospital, Melbourne, Victoria, Australia.; Department of Paediatrics, Murdoch Children's Research Institute, University of Melbourne, Melbourne, Victoria, Australia.; Mayoh C; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.; Wong M; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.; Barahona P; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; Ajuyah P; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; Senapati A; Kids Cancer Centre, Sydney Children's Hospital, Sydney, New South Wales, Australia.; Nagabushan S; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.; Kids Cancer Centre, Sydney Children's Hospital, Sydney, New South Wales, Australia.; Sherstyuk A; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; Altekoester AK; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; Fuentes-Bolanos NA; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.; Kids Cancer Centre, Sydney Children's Hospital, Sydney, New South Wales, Australia.; Yeung V; Kids Cancer Centre, Sydney Children's Hospital, Sydney, New South Wales, Australia.; Sullivan A; Oncology Services Group, Children's Health Queensland Hospital & Health Service, Brisbane, Queensland, Australia.; Omer N; Oncology Services Group, Children's Health Queensland Hospital & Health Service, Brisbane, Queensland, Australia.; Frazer Institute, Faculty of Medicine, The University of Queensland, Brisbane, Queensland, Australia.; Diamond Y; Children's Cancer Centre, Royal Children's Hospital, Melbourne, Victoria, Australia.; Jessop S; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; Michael Rice Centre for Haematology and Oncology, Women's and Children's Hospital, Adelaide, South Australia, Australia.; Battaglia L; Children's Cancer Centre, Monash Children's Hospital, Melbourne, Victoria, Australia.; Zhukova N; Children's Cancer Centre, Monash Children's Hospital, Melbourne, Victoria, Australia.; Centre for Cancer Research, Hudson Institute of Medical Research, Melbourne, Victoria, Australia.; Department of Paediatrics, School of Clinical Sciences at Monash Health, Faculty of Medicine, Nursing and Health Sciences, Monash University, Melbourne, Victoria, Australia.; Cui L; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; Lin A; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; Gifford AJ; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.; Anatomical Pathology, NSW Health Pathology, Prince of Wales Hospital, Sydney, New South Wales, Australia.; Fleuren EDG; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.; Dalla-Pozza L; Cancer Centre for Children, The Children's Hospital at Westmead, Sydney, New South Wales, Australia.; Moore AS; Oncology Services Group, Children's Health Queensland Hospital & Health Service, Brisbane, Queensland, Australia.; Khaw SL; Children's Cancer Centre, Royal Children's Hospital, Melbourne, Victoria, Australia.; Department of Paediatrics, Murdoch Children's Research Institute, University of Melbourne, Melbourne, Victoria, Australia.; Eisenstat DD; Children's Cancer Centre, Royal Children's Hospital, Melbourne, Victoria, Australia.; Department of Paediatrics, Murdoch Children's Research Institute, University of Melbourne, Melbourne, Victoria, Australia.; Gottardo NG; Department of Paediatric and Adolescent Oncology and Haematology, Perth Children's Hospital, Perth, Western Australia, Australia.; Telethon Kids Cancer Centre, Telethon Kids Institute, University of Western Australia, Perth, Western Australia, Australia.; Wood PJ; Children's Cancer Centre, Monash Children's Hospital, Melbourne, Victoria, Australia.; Centre for Cancer Research, Hudson Institute of Medical Research, Melbourne, Victoria, Australia.; Department of Paediatrics, School of Clinical Sciences at Monash Health, Faculty of Medicine, Nursing and Health Sciences, Monash University, Melbourne, Victoria, Australia.; Tapp H; Michael Rice Centre for Haematology and Oncology, Women's and Children's Hospital, Adelaide, South Australia, Australia.; Alvaro F; Children's Cancer and Blood Disorders, John Hunter Children's Hospital, University of Newcastle, Newcastle, New South Wales, Australia.; McCowage G; Cancer Centre for Children, The Children's Hospital at Westmead, Sydney, New South Wales, Australia.; Nicholls W; Oncology Services Group, Children's Health Queensland Hospital & Health Service, Brisbane, Queensland, Australia.; Frazer Institute, Faculty of Medicine, The University of Queensland, Brisbane, Queensland, Australia.; Hansford JR; Children's Cancer Centre, Royal Children's Hospital, Melbourne, Victoria, Australia.; Department of Paediatrics, Murdoch Children's Research Institute, University of Melbourne, Melbourne, Victoria, Australia.; Michael Rice Centre for Haematology and Oncology, Women's and Children's Hospital, Adelaide, South Australia, Australia.; Department of Paediatrics, Faculty of Medicine, Dentistry and Health Sciences, University of Melbourne, Melbourne, Victoria, Australia.; South Australia Health and Medical Research Institute, Adelaide, South Australia, Australia.; South Australian immunoGENomics Cancer Institute, University of Adelaide, Adelaide, South Australia, Australia.; Manoharan N; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.; Kids Cancer Centre, Sydney Children's Hospital, Sydney, New South Wales, Australia.; Kotecha RS; Department of Paediatric and Adolescent Oncology and Haematology, Perth Children's Hospital, Perth, Western Australia, Australia.; Telethon Kids Cancer Centre, Telethon Kids Institute, University of Western Australia, Perth, Western Australia, Australia.; Curtin Medical School, Curtin University, Perth, Western Australia, Australia.; Mateos MK; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.; Kids Cancer Centre, Sydney Children's Hospital, Sydney, New South Wales, Australia.; Lock RB; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.; UNSW Centre for Childhood Cancer Research, UNSW Sydney, Sydney, New South Wales, Australia.; Tyrrell V; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.; Haber M; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.; Trahair TN; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.; Kids Cancer Centre, Sydney Children's Hospital, Sydney, New South Wales, Australia.; Cowley MJ; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.; Ekert PG; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.; Department of Paediatrics, Murdoch Children's Research Institute, University of Melbourne, Melbourne, Victoria, Australia.; Cancer Immunology Program, Peter MacCallum Cancer Centre, Melbourne, Victoria, Australia.; Marshall GM; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.; Kids Cancer Centre, Sydney Children's Hospital, Sydney, New South Wales, Australia.; Ziegler DS; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia. d.ziegler@unsw.edu.au.; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia. d.ziegler@unsw.edu.au.; Kids Cancer Centre, Sydney Children's Hospital, Sydney, New South Wales, Australia. d.ziegler@unsw.edu.au.
- Source
- Publisher: Nature Publishing Company Country of Publication: United States NLM ID: 9502015 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1546-170X (Electronic) Linking ISSN: 10788956 NLM ISO Abbreviation: Nat Med Subsets: MEDLINE
- Subject
- Language
- English
Recent research showed that precision medicine can identify new treatment strategies for patients with childhood cancers. However, it is unclear which patients will benefit most from precision-guided treatment (PGT). Here we report consecutive data from 384 patients with high-risk pediatric cancer (with an expected cure rate of less than 30%) who had at least 18 months of follow-up on the ZERO Childhood Cancer Precision Medicine Program PRecISion Medicine for Children with Cancer (PRISM) trial. A total of 256 (67%) patients received PGT recommendations and 110 (29%) received a recommended treatment. PGT resulted in a 36% objective response rate and improved 2-year progression-free survival compared with standard of care (26% versus 12%; P = 0.049) or targeted agents not guided by molecular findings (26% versus 5.2%; P = 0.003). PGT based on tier 1 evidence, PGT targeting fusions or commenced before disease progression had the greatest clinical benefit. Our data show that PGT informed by comprehensive molecular profiling significantly improves outcomes for children with high-risk cancers. ClinicalTrials.gov registration: NCT03336931.
(© 2024. The Author(s).)