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@ARTICLE{Giesen:186669,
      author       = {N. Giesen$^*$ and M. Chatterjee and C. Scheid and A. M.
                      Poos and B. Besemer and K. Miah$^*$ and A. Benner$^*$ and N.
                      Becker and T. Moehler and I. Metzler and C. Khandanpour and
                      A. Seidel-Glaetzer and K. Trautmann-Grill and K. M. Kortüm
                      and C. Müller-Tidow and G. Mechtersheimer and B. Goeppert
                      and A. Stenzinger and N. Weinhold and H. Goldschmidt and K.
                      C. Weisel and M.-S. Raab$^*$},
      title        = {{A} phase {II} clinical trial of combined {BRAF}/{MEK}
                      inhibition for {BRAFV}600{E}-mutated multiple myeloma.},
      journal      = {Blood},
      volume       = {141},
      number       = {14},
      issn         = {0006-4971},
      address      = {Washington, DC},
      publisher    = {American Society of Hematology},
      reportid     = {DKFZ-2023-00038},
      pages        = {1685-1690},
      year         = {2023},
      note         = {#EA:A360#LA:A360# / 2023 Apr 6;141(14):1685-1690},
      abstract     = {Activating BRAF mutations are found in a small subset of
                      patients with newly diagnosed multiple myeloma but
                      prevalence increases in late stage, refractory disease, and
                      are associated with adverse outcome. This prospective
                      single-arm, open-label, multicenter phase II trial assessed
                      the efficacy and safety of combined BRAF/MEK inhibition,
                      using encorafenib and binimetinib, in patients with
                      relapsed/refractory multiple myeloma (RRMM) carrying a
                      BRAFV600E mutation (ClinicalTrials.gov identifier:
                      NCT02834364). Patients received 450 mg encorafenib once
                      daily and binimetinib 45 mg twice daily. The primary end
                      point was the overall response rate achieved within the
                      first year after start of treatment according to IMWG
                      criteria. Twelve RRMM patients with a median of 5 prior
                      lines of therapy were enrolled. The overall response rate
                      was $83.3\%$ with 10 patients achieving at least a partial
                      response. The median progression-free survival (PFS) was 5.6
                      months and overall survival was $55\%$ at 24 months.
                      Emerging resistance to therapy was driven by RAS mutations
                      and structural variants involving the BRAF locus. This is
                      the first prospective clinical trial to demonstrate that
                      combined BRAF/MEK inhibition is highly effective in patients
                      with BRAFV600E mutated RRMM and represents a successful
                      targeted precision medicine approach in this disease.},
      cin          = {A360 / C060},
      ddc          = {610},
      cid          = {I:(DE-He78)A360-20160331 / I:(DE-He78)C060-20160331},
      pnm          = {311 - Zellbiologie und Tumorbiologie (POF4-311)},
      pid          = {G:(DE-HGF)POF4-311},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:36608320},
      doi          = {10.1182/blood.2022017789},
      url          = {https://inrepo02.dkfz.de/record/186669},
}