PAX5 mutations occur frequently in adult B-cell progenitor acute lymphoblastic leukemia and PAX5 haploinsufficiency is associated with BCR-ABL1 and TCF3-PBX1 fusion genes: a GRAALL study

Leukemia. 2009 Nov;23(11):1989-98. doi: 10.1038/leu.2009.135. Epub 2009 Jul 9.

Abstract

Adult and child B-cell progenitor acute lymphoblastic leukemia (BCP-ALL) differ in terms of incidence and prognosis. These disparities are mainly due to the molecular abnormalities associated with these two clinical entities. A genome-wide analysis using oligo SNP arrays recently demonstrated that PAX5 (paired-box domain 5) is the main target of somatic mutations in childhood BCP-ALL being altered in 38.9% of the cases. We report here the most extensive analysis of alterations of PAX5 coding sequence in 117 adult BCP-ALL patients in the unique clinical protocol GRAALL-2003/GRAAPH-2003. Our study demonstrates that PAX5 is mutated in 34% of adult BCP-ALL, mutations being partial or complete deletion, partial or complete amplification, point mutation or fusion gene. PAX5 alterations are heterogeneous consisting in complete loss in 17%, focal deletions in 10%, point mutations in 7% and translocations in 1% of the cases. PAX5 complete loss and PAX5 point mutations differ. PAX5 complete loss seems to be a secondary event and is significantly associated with BCR-ABL1 or TCF3-PBX1 fusion genes and a lower white blood cell count.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Antineoplastic Agents / therapeutic use
  • Basic Helix-Loop-Helix Transcription Factors / genetics*
  • Benzamides
  • Clinical Trials, Phase II as Topic
  • DNA-Binding Proteins / genetics*
  • Fusion Proteins, bcr-abl / genetics*
  • Gene Dosage
  • Gene Rearrangement, T-Lymphocyte / genetics
  • Genomics
  • Haplotypes
  • Humans
  • Imatinib Mesylate
  • Immunoglobulin Heavy Chains / genetics
  • Immunophenotyping
  • Middle Aged
  • Multicenter Studies as Topic
  • PAX5 Transcription Factor / genetics*
  • Piperazines / therapeutic use
  • Point Mutation
  • Pre-B-Cell Leukemia Transcription Factor 1
  • Precursor B-Cell Lymphoblastic Leukemia-Lymphoma / drug therapy
  • Precursor B-Cell Lymphoblastic Leukemia-Lymphoma / genetics*
  • Prognosis
  • Prospective Studies
  • Proto-Oncogene Proteins / genetics*
  • Pyrimidines / therapeutic use
  • Young Adult

Substances

  • Antineoplastic Agents
  • Basic Helix-Loop-Helix Transcription Factors
  • Benzamides
  • DNA-Binding Proteins
  • Immunoglobulin Heavy Chains
  • PAX5 Transcription Factor
  • PAX5 protein, human
  • Piperazines
  • Pre-B-Cell Leukemia Transcription Factor 1
  • Proto-Oncogene Proteins
  • Pyrimidines
  • TCF3 protein, human
  • PBX1 protein, human
  • Imatinib Mesylate
  • Fusion Proteins, bcr-abl