Precision-guided treatment in high-risk pediatric cancers.

Autor: 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.
Jazyk: angličtina
Zdroj: Nature medicine [Nat Med] 2024 Jul; Vol. 30 (7), pp. 1913-1922. Date of Electronic Publication: 2024 Jun 06.
DOI: 10.1038/s41591-024-03044-0
Abstrakt: 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).)
Databáze: MEDLINE
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