Whole-exome sequencing identifies somatic mutations of BCOR in acute myeloid leukemia with normal karyotype.

نویسندگان

  • Vera Grossmann
  • Enrico Tiacci
  • Antony B Holmes
  • Alexander Kohlmann
  • Maria Paola Martelli
  • Wolfgang Kern
  • Ariele Spanhol-Rosseto
  • Hans-Ulrich Klein
  • Martin Dugas
  • Sonja Schindela
  • Vladimir Trifonov
  • Susanne Schnittger
  • Claudia Haferlach
  • Renato Bassan
  • Victoria A Wells
  • Orietta Spinelli
  • Joseph Chan
  • Roberta Rossi
  • Stefano Baldoni
  • Luca De Carolis
  • Katharina Goetze
  • Hubert Serve
  • Rudolf Peceny
  • Karl-Anton Kreuzer
  • Daniel Oruzio
  • Giorgina Specchia
  • Francesco Di Raimondo
  • Francesco Fabbiano
  • Marco Sborgia
  • Arcangelo Liso
  • Laurent Farinelli
  • Alessandro Rambaldi
  • Laura Pasqualucci
  • Raul Rabadan
  • Torsten Haferlach
  • Brunangelo Falini
چکیده

Among acute myeloid leukemia (AML) patients with a normal karyotype (CN-AML), NPM1 and CEBPA mutations define World Health Organization 2008 provisional entities accounting for approximately 60% of patients, but the remaining 40% are molecularly poorly characterized. Using whole-exome sequencing of one CN-AML patient lacking mutations in NPM1, CEBPA, FLT3-ITD, IDH1, and MLL-PTD, we newly identified a clonal somatic mutation in BCOR (BCL6 corepressor), a gene located on chromosome Xp11.4. Further analyses of 553 AML patients showed that BCOR mutations occurred in 3.8% of unselected CN-AML patients and represented a substantial fraction (17.1%) of CN-AML patients showing the same genotype as the AML index patient subjected to whole-exome sequencing. BCOR somatic mutations were: (1) disruptive events similar to the germline BCOR mutations causing the oculo-facio-cardio-dental genetic syndrome; (2) associated with decreased BCOR mRNA levels, absence of full-length BCOR, and absent or low expression of a truncated BCOR protein; (3) virtually mutually exclusive with NPM1 mutations; and (4) frequently associated with DNMT3A mutations, suggesting cooperativity among these genetic alterations. Finally, BCOR mutations tended to be associated with an inferior outcome in a cohort of 422 CN-AML patients (25.6% vs 56.7% overall survival at 2 years; P = .032). Our results for the first time implicate BCOR in CN-AML pathogenesis.

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MYELOID NEOPLASIA Whole-exome sequencing identifies somatic mutations of BCOR in acute myeloid leukemia with normal karyotype

*Vera Grossmann,1 *Enrico Tiacci,2 Antony B. Holmes,3 Alexander Kohlmann,1 Maria Paola Martelli,2 Wolfgang Kern,1 Ariele Spanhol-Rosseto,2 Hans-Ulrich Klein,4 Martin Dugas,4 Sonja Schindela,1 Vladimir Trifonov,3 Susanne Schnittger,1 Claudia Haferlach,1 Renato Bassan,5 Victoria A. Wells,6 Orietta Spinelli,5 Joseph Chan,3 Roberta Rossi,2 Stefano Baldoni,2 Luca De Carolis,2 Katharina Goetze,7 Hube...

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عنوان ژورنال:
  • Blood

دوره 118 23  شماره 

صفحات  -

تاریخ انتشار 2011