SETBP1 mutations occur in 9% of MDS/MPN and in 4% of MPN cases and are strongly associated with atypical CML, monosomy 7, isochromosome i(17)(q10), ASXL1 and CBL mutations

Leukemia. 2013 Sep;27(9):1852-60. doi: 10.1038/leu.2013.133. Epub 2013 Apr 30.

Abstract

Chronic myeloid malignancies are categorized to the three main categories myeloproliferative neoplasms (MPNs), myelodysplastic syndromes (MDSs) and MDS/MPN overlap. So far, no specific genetic alteration profiles have been identified in the MDS/MPN overlap category. Recent studies identified mutations in SET-binding protein 1 (SETBP1) as novel marker in myeloid malignancies, especially in atypical chronic myeloid leukemia (aCML) and related diseases. We analyzed SETBP1 in 1 130 patients with MPN and MDS/MPN overlap and found mutation frequencies of 3.8% and 9.4%, respectively. In particular, there was a high frequency of SETBP1 mutation in aCML (19/60; 31.7%) and MDS/MPN unclassifiable (MDS/MPN, U; 20/240; 9.3%). SETBP1 mutated (SETBP1mut) patients showed significantly higher white blood cell counts and lower platelet counts and hemoglobin levels than SETBP1 wild-type patients. Cytomorphologic evaluation revealed a more dysplastic phenotype in SETBP1mut cases as compared with wild-type cases. We confirm a significant association of SETBP1mut with -7 and isochromosome i(17)(q10). Moreover, SETBP1mut were strongly associated with ASXL1 and CBL mutations (P<0.001 for both) and were mutually exclusive of JAK2 and TET2 mutations. In conclusion, SETBP1mut add an important new diagnostic marker for MDS/MPN and in particular for aCML.

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Bone Marrow / pathology
  • Carrier Proteins / genetics*
  • Chromosome Deletion*
  • Chromosomes, Human, Pair 7
  • Female
  • Humans
  • Isochromosomes*
  • Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative / diagnosis
  • Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative / genetics
  • Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative / mortality
  • Male
  • Middle Aged
  • Mutation Rate
  • Mutation*
  • Myelodysplastic Syndromes / diagnosis
  • Myelodysplastic Syndromes / genetics*
  • Myelodysplastic Syndromes / mortality
  • Myelodysplastic-Myeloproliferative Diseases / diagnosis
  • Myelodysplastic-Myeloproliferative Diseases / genetics*
  • Myelodysplastic-Myeloproliferative Diseases / mortality
  • Nuclear Proteins / genetics*
  • Proto-Oncogene Proteins c-cbl / genetics*
  • Repressor Proteins / genetics*
  • Young Adult

Substances

  • ASXL1 protein, human
  • Carrier Proteins
  • Nuclear Proteins
  • Repressor Proteins
  • SETBP1 protein, human
  • Proto-Oncogene Proteins c-cbl

Supplementary concepts

  • Chromosome 7, monosomy