Pediatric-inspired intensified therapy of adult T-ALL reveals the favorable outcome of NOTCH1/FBXW7 mutations, but not of low ERG/BAALC expression: a GRAALL study

Blood. 2011 Nov 10;118(19):5099-107. doi: 10.1182/blood-2011-02-334219. Epub 2011 Aug 11.

Abstract

Despite recent progress in the understanding of acute lymphoblastic leukemia (T-ALL) oncogenesis, few markers are sufficiently frequent in large subgroups to allow their use in therapeutic stratification. Low ERG and BAALC expression (E/B(low)) and NOTCH1/FBXW7 (N/F) mutations have been proposed as powerful prognostic markers in large cohorts of adult T-ALL. We therefore compared the predictive prognostic value of N/F mutations versus E/B(low) in 232 adult T-ALLs enrolled in the LALA-94 and Group for Research on Adult Acute Lymphoblastic Leukemia (GRAALL) protocols. The outcome of T-ALLs treated in the pediatric-inspired GRAALL trials was significantly superior to the LALA-94 trial. Overall, 43% and 69% of adult T-ALL patients were classified as E/B(low) and N/F mutated, respectively. Strikingly, the good prognosis of N/F mutated patients was stronger in more intensively treated, pediatric-inspired GRAALL patients. The E/B expression level did not influence the prognosis in any subgroup. N/F mutation status and the GRAALL trial were the only 2 independent factors that correlated with longer overall survival by multivariate analysis. This study demonstrates that the N/F mutational status and treatment protocol are major outcome determinants for adults with T-ALL, the benefit of pediatric inspired protocols being essentially restricted to the N/F mutated subgroup.

Publication types

  • Comparative Study
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Cell Cycle Proteins / genetics*
  • Child
  • Clinical Protocols
  • Disease-Free Survival
  • F-Box Proteins / genetics*
  • F-Box-WD Repeat-Containing Protein 7
  • Female
  • Gene Expression
  • Humans
  • Kaplan-Meier Estimate
  • Leukemia-Lymphoma, Adult T-Cell / classification
  • Leukemia-Lymphoma, Adult T-Cell / drug therapy*
  • Leukemia-Lymphoma, Adult T-Cell / genetics*
  • Male
  • Middle Aged
  • Mutation*
  • Neoplasm Proteins / genetics*
  • Predictive Value of Tests
  • Prognosis
  • Prospective Studies
  • Receptor, Notch1 / genetics*
  • Trans-Activators / genetics*
  • Transcriptional Regulator ERG
  • Treatment Outcome
  • Ubiquitin-Protein Ligases / genetics*
  • Young Adult

Substances

  • BAALC protein, human
  • Cell Cycle Proteins
  • ERG protein, human
  • F-Box Proteins
  • F-Box-WD Repeat-Containing Protein 7
  • FBXW7 protein, human
  • NOTCH1 protein, human
  • Neoplasm Proteins
  • Receptor, Notch1
  • Trans-Activators
  • Transcriptional Regulator ERG
  • Ubiquitin-Protein Ligases