Precision-guided treatment in high-risk pediatric cancers.
Nat Med
; 30(7): 1913-1922, 2024 Jul.
Article
in 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.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Precision Medicine
/
Neoplasms
Limits:
Adolescent
/
Child
/
Child, preschool
/
Female
/
Humans
/
Infant
/
Male
Language:
En
Journal:
Nat Med
/
Nat. med
/
Nature medicine
Journal subject:
BIOLOGIA MOLECULAR
/
MEDICINA
Year:
2024
Document type:
Article
Affiliation country:
Australia
Country of publication:
Estados Unidos