The coding genome of splenic marginal zone lymphoma: activation of NOTCH2 and other pathways regulating marginal zone development

نویسندگان

  • Davide Rossi
  • Vladimir Trifonov
  • Marco Fangazio
  • Alessio Bruscaggin
  • Silvia Rasi
  • Valeria Spina
  • Sara Monti
  • Tiziana Vaisitti
  • Francesca Arruga
  • Rosella Famà
  • Carmela Ciardullo
  • Mariangela Greco
  • Stefania Cresta
  • Daniela Piranda
  • Antony Holmes
  • Giulia Fabbri
  • Monica Messina
  • Andrea Rinaldi
  • Jiguang Wang
  • Claudio Agostinelli
  • Pier Paolo Piccaluga
  • Marco Lucioni
  • Fabrizio Tabbò
  • Roberto Serra
  • Silvia Franceschetti
  • Clara Deambrogi
  • Giulia Daniele
  • Valter Gattei
  • Roberto Marasca
  • Fabio Facchetti
  • Luca Arcaini
  • Giorgio Inghirami
  • Francesco Bertoni
  • Stefano A. Pileri
  • Silvia Deaglio
  • Robin Foà
  • Riccardo Dalla-Favera
  • Laura Pasqualucci
  • Raul Rabadan
  • Gianluca Gaidano
چکیده

Splenic marginal zone lymphoma (SMZL) is a B cell malignancy of unknown pathogenesis, and thus an orphan of targeted therapies. By integrating whole-exome sequencing and copy-number analysis, we show that the SMZL exome carries at least 30 nonsilent gene alterations. Mutations in NOTCH2, a gene required for marginal-zone (MZ) B cell development, represent the most frequent lesion in SMZL, accounting for ∼20% of cases. All NOTCH2 mutations are predicted to cause impaired degradation of the NOTCH2 protein by eliminating the C-terminal PEST domain, which is required for proteasomal recruitment. Among indolent B cell lymphoproliferative disorders, NOTCH2 mutations are restricted to SMZL, thus representing a potential diagnostic marker for this lymphoma type. In addition to NOTCH2, other modulators or members of the NOTCH pathway are recurrently targeted by genetic lesions in SMZL; these include NOTCH1, SPEN, and DTX1. We also noted mutations in other signaling pathways normally involved in MZ B cell development, suggesting that deregulation of MZ B cell development pathways plays a role in the pathogenesis of ∼60% SMZL. These findings have direct implications for the treatment of SMZL patients, given the availability of drugs that can target NOTCH, NF-κB, and other pathways deregulated in this disease.

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عنوان ژورنال:

دوره 209  شماره 

صفحات  -

تاریخ انتشار 2012