Genetic discoveries and treatment advances in neuroblastoma.
Curr Opin Pediatr
; 28(1): 19-25, 2016 Feb.
Article
em En
| MEDLINE
| ID: mdl-26576010
ABSTRACT
PURPOSE OF REVIEW Major advances in our understanding of the genetic basis of neuroblastoma, and the role somatic alterations play in driving tumor growth, have led to improvements in risk-stratified therapy and have provided the rationale for targeted therapies. In this review, we highlight current risk-based treatment approaches and discuss the opportunities and challenges of translating recent genomic discoveries into the clinic. RECENT FINDINGS:
Significant progress in the treatment of neuroblastoma has been realized using risk-based treatment strategies. Outcome has improved for all patients, including those classified as high-risk, although survival remains poor for this cohort. Integration of whole-genome DNA copy number and comprehensive molecular profiles into neuroblastoma classification systems will allow more precise prognostication and refined treatment assignment. Promising treatments that include targeted systemic radiotherapy, pathway-targeted small molecules, and therapy targeted at cell surface molecules are being evaluated in clinical trials, and recent genomic discoveries in relapsed tumor samples have led to the identification of new actionable mutations.SUMMARY:
The integration of refined treatment stratification based on whole-genome profiles with therapeutics that target the molecular drivers of malignant behavior in neuroblastoma has the potential to dramatically improve survival, with decreased toxicity.
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Terapia de Alvo Molecular
/
Neuroblastoma
Tipo de estudo:
Etiology_studies
/
Prognostic_studies
/
Risk_factors_studies
Limite:
Child
/
Humans
Idioma:
En
Revista:
Curr Opin Pediatr
Assunto da revista:
PEDIATRIA
Ano de publicação:
2016
Tipo de documento:
Article
País de afiliação:
Estados Unidos