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Precision-guided treatment in high-risk pediatric cancers.
Lau, Loretta M S; Khuong-Quang, Dong-Anh; Mayoh, Chelsea; Wong, Marie; Barahona, Paulette; Ajuyah, Pamela; Senapati, Akanksha; Nagabushan, Sumanth; Sherstyuk, Alexandra; Altekoester, Ann-Kristin; Fuentes-Bolanos, Noemi A; Yeung, Veronica; Sullivan, Ashleigh; Omer, Natacha; Diamond, Yonatan; Jessop, Sophie; Battaglia, Lauren; Zhukova, Nataliya; Cui, Louise; Lin, Angela; Gifford, Andrew J; Fleuren, Emmy D G; Dalla-Pozza, Luciano; Moore, Andrew S; Khaw, Seong-Lin; Eisenstat, David D; Gottardo, Nicholas G; Wood, Paul J; Tapp, Heather; Alvaro, Frank; McCowage, Geoffrey; Nicholls, Wayne; Hansford, Jordan R; Manoharan, Neevika; Kotecha, Rishi S; Mateos, Marion K; Lock, Richard B; Tyrrell, Vanessa; Haber, Michelle; Trahair, Toby N; Cowley, Mark J; Ekert, Paul G; Marshall, Glenn M; Ziegler, David S.
Afiliação
  • Lau LMS; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.
  • Khuong-Quang DA; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.
  • Mayoh C; Kids Cancer Centre, Sydney Children's Hospital, Sydney, New South Wales, Australia.
  • Wong M; Children's Cancer Centre, Royal Children's Hospital, Melbourne, Victoria, Australia.
  • Barahona P; Department of Paediatrics, Murdoch Children's Research Institute, University of Melbourne, Melbourne, Victoria, Australia.
  • Ajuyah P; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.
  • Senapati A; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.
  • Nagabushan S; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.
  • Sherstyuk A; School of Clinical Medicine, UNSW Medicine & Health, 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.
  • Yeung V; Kids Cancer Centre, Sydney Children's Hospital, Sydney, New South Wales, Australia.
  • Sullivan A; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.
  • Omer N; Kids Cancer Centre, Sydney Children's Hospital, Sydney, New South Wales, Australia.
  • Diamond Y; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.
  • Jessop S; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.
  • Battaglia L; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.
  • Zhukova N; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.
  • Cui L; Kids Cancer Centre, Sydney Children's Hospital, Sydney, New South Wales, Australia.
  • Lin A; Kids Cancer Centre, Sydney Children's Hospital, Sydney, New South Wales, Australia.
  • Gifford AJ; Oncology Services Group, Children's Health Queensland Hospital & Health Service, Brisbane, Queensland, Australia.
  • Fleuren EDG; Oncology Services Group, Children's Health Queensland Hospital & Health Service, Brisbane, Queensland, Australia.
  • Dalla-Pozza L; Frazer Institute, Faculty of Medicine, The University of Queensland, Brisbane, Queensland, Australia.
  • Moore AS; Children's Cancer Centre, Royal Children's Hospital, Melbourne, Victoria, Australia.
  • Khaw SL; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.
  • Eisenstat DD; Michael Rice Centre for Haematology and Oncology, Women's and Children's Hospital, Adelaide, South Australia, Australia.
  • Gottardo NG; Children's Cancer Centre, Monash Children's Hospital, Melbourne, Victoria, Australia.
  • Wood PJ; Children's Cancer Centre, Monash Children's Hospital, Melbourne, Victoria, Australia.
  • Tapp H; Centre for Cancer Research, Hudson Institute of Medical Research, Melbourne, Victoria, Australia.
  • Alvaro F; Department of Paediatrics, School of Clinical Sciences at Monash Health, Faculty of Medicine, Nursing and Health Sciences, Monash University, Melbourne, Victoria, Australia.
  • McCowage G; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.
  • Nicholls W; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.
  • Hansford JR; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.
  • Manoharan N; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.
  • Kotecha RS; Anatomical Pathology, NSW Health Pathology, Prince of Wales Hospital, Sydney, New South Wales, Australia.
  • Mateos MK; Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.
  • Lock RB; School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.
  • Tyrrell V; Cancer Centre for Children, The Children's Hospital at Westmead, Sydney, New South Wales, Australia.
  • Haber M; Oncology Services Group, Children's Health Queensland Hospital & Health Service, Brisbane, Queensland, Australia.
  • Trahair TN; Children's Cancer Centre, Royal Children's Hospital, Melbourne, Victoria, Australia.
  • Cowley MJ; Department of Paediatrics, Murdoch Children's Research Institute, University of Melbourne, Melbourne, Victoria, Australia.
  • Ekert PG; Children's Cancer Centre, Royal Children's Hospital, Melbourne, Victoria, Australia.
  • Marshall GM; Department of Paediatrics, Murdoch Children's Research Institute, University of Melbourne, Melbourne, Victoria, Australia.
  • Ziegler DS; Department of Paediatric and Adolescent Oncology and Haematology, Perth Children's Hospital, Perth, Western Australia, Australia.
Nat Med ; 30(7): 1913-1922, 2024 Jul.
Article em En | MEDLINE | ID: mdl-38844796
ABSTRACT
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.
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Medicina de Precisão / Neoplasias Idioma: En Ano de publicação: 2024 Tipo de documento: Article
Texto completo
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Medicina de Precisão / Neoplasias Idioma: En Ano de publicação: 2024 Tipo de documento: Article