Genetic analysis of patients with leukemic transformation of myeloproliferative neoplasms shows recurrent SRSF2 mutations that are associated with adverse outcome

Blood. 2012 May 10;119(19):4480-5. doi: 10.1182/blood-2011-11-390252. Epub 2012 Mar 19.

Abstract

Leukemic transformation (LT) of myeloproliferative neoplasms (MPNs) is associated with a poor prognosis and resistance to therapy. Although previous candidate genetic studies have identified mutations in MPN patients who develop acute leukemia, the complement of genetic abnormalities in MPN patients who undergo LT is not known nor have specific molecular abnormalities been shown to have clinical relevance in this setting. We performed high-throughput resequencing of 22 genes in 53 patients with LT after MPN to characterize the frequency of known myeloid mutations in this entity. In addition to JAK2 and TET2 mutations, which occur commonly in LT after MPN, we identified recurrent mutations in the serine/arginine-rich splicing factor 2 (SRSF2) gene (18.9%) in acute myeloid leukemia (AML) transformed from MPNs. SRSF2 mutations are more common in AML derived from MPNs compared with LT after myelodysplasia (4.8%) or de novo AML (5.6%), respectively (P=.05). Importantly, SRSF2 mutations are associated with worsened overall survival in MPN patients who undergo LT in univariate (P=.03; HR, 2.77; 95% CI, 1.10-7.00) and multivariate analysis (P<.05; HR, 2.11; 95% CI, 1.01-4.42). These data suggest that SRSF2 mutations contribute to the pathogenesis of LT and may guide novel therapeutic approaches for MPN patients who undergo LT.

Publication types

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

MeSH terms

  • Base Sequence
  • Cell Transformation, Neoplastic / genetics*
  • Cell Transformation, Neoplastic / pathology
  • Cohort Studies
  • DNA Mutational Analysis
  • Disease Progression
  • Gene Frequency
  • Hematologic Neoplasms / diagnosis
  • Hematologic Neoplasms / genetics*
  • Hematologic Neoplasms / mortality
  • Hematologic Neoplasms / pathology
  • Humans
  • Leukemia / diagnosis
  • Leukemia / genetics
  • Leukemia / mortality
  • Leukemia / pathology*
  • Mutation / physiology
  • Myeloproliferative Disorders / diagnosis*
  • Myeloproliferative Disorders / genetics*
  • Myeloproliferative Disorders / mortality
  • Myeloproliferative Disorders / pathology
  • Nuclear Proteins / genetics*
  • Prognosis
  • Ribonucleoproteins / genetics*
  • Serine-Arginine Splicing Factors
  • Spliceosomes / genetics
  • Spliceosomes / metabolism

Substances

  • Nuclear Proteins
  • Ribonucleoproteins
  • SRSF2 protein, human
  • Serine-Arginine Splicing Factors