PAX5-driven subtypes of B-progenitor acute lymphoblastic leukemia

Nat Genet. 2019 Feb;51(2):296-307. doi: 10.1038/s41588-018-0315-5. Epub 2019 Jan 14.

Abstract

Recent genomic studies have identified chromosomal rearrangements defining new subtypes of B-progenitor acute lymphoblastic leukemia (B-ALL), however many cases lack a known initiating genetic alteration. Using integrated genomic analysis of 1,988 childhood and adult cases, we describe a revised taxonomy of B-ALL incorporating 23 subtypes defined by chromosomal rearrangements, sequence mutations or heterogeneous genomic alterations, many of which show marked variation in prevalence according to age. Two subtypes have frequent alterations of the B lymphoid transcription-factor gene PAX5. One, PAX5alt (7.4%), has diverse PAX5 alterations (rearrangements, intragenic amplifications or mutations); a second subtype is defined by PAX5 p.Pro80Arg and biallelic PAX5 alterations. We show that p.Pro80Arg impairs B lymphoid development and promotes the development of B-ALL with biallelic Pax5 alteration in vivo. These results demonstrate the utility of transcriptome sequencing to classify B-ALL and reinforce the central role of PAX5 as a checkpoint in B lymphoid maturation and leukemogenesis.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acute Disease
  • Adolescent
  • Adult
  • Aged
  • Animals
  • Child
  • Child, Preschool
  • Chromosomes / genetics
  • Female
  • Gene Rearrangement / genetics
  • Humans
  • Infant
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Middle Aged
  • Mutation / genetics
  • PAX5 Transcription Factor / genetics*
  • Precursor B-Cell Lymphoblastic Leukemia-Lymphoma / genetics*
  • Transcriptome / genetics
  • Young Adult

Substances

  • PAX5 Transcription Factor
  • PAX5 protein, human