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@ARTICLE{Menzer:304097,
      author       = {C. Menzer and S. Dugas-Breit and M. Dugas and C. U. Blank
                      and E. J. Groen and I. Reijers and M. Schlaak and J. Eckardt
                      and K. P. M. Suijkerbuijk and L. Zimmer$^*$ and D. B.
                      Johnson and C. Franklin and F. Meiss and B. Schilling and F.
                      Meier and R. Gutzmer and K.-M. Thoms and T. Haalck and M.
                      Müller$^*$ and A. Kopp-Schneider$^*$ and M. S. Carlino and
                      G. V. Long and A. M. Menzies and A. A. M. van der Veldt and
                      J. W. B. de Groot and T. Eigentler and M. Stevense-den Boer
                      and C. Pföhler and K. Herbschleb and J. C. Hassel},
      title        = {{T}argeted therapy for rare {BRAF}-mutated melanoma:
                      {U}pdated multicenter analysis and launch of a publicly
                      accessible online outcome database.},
      journal      = {European journal of cancer},
      volume       = {228},
      issn         = {0959-8049},
      address      = {Amsterdam [u.a.]},
      publisher    = {Elsevier},
      reportid     = {DKFZ-2025-01765},
      pages        = {115703},
      year         = {2025},
      abstract     = {While BRAF-/MEK-inhibitor therapy is well established in
                      V600E/K-mutated melanoma, the efficacy in advanced melanoma
                      with rare BRAF mutations remains uncertain. This is an
                      updated analysis of an international data collection
                      including 49 new patients, accompanied by development of a
                      publicly accessible global database.A retrospective analysis
                      was conducted at 20 international cancer centers, evaluating
                      143 patients with rare BRAF V600 (V600-nonE/K; 48 $\%)$ and
                      non-V600 (52 $\%)$ mutations. Treatments included BRAF/MEK
                      inhibitor combination therapy (BRAFi/MEKi) and the
                      respective monotherapies. Clinical outcomes concerning
                      overall response rate (ORR), progression-free (PFS), and
                      overall survival (OS) were collected.Included patients had a
                      median age of 65 years (range 20-93), 101 (71 $\%)$ were
                      male. Most patients (n = 92, 64 $\%)$ received BRAFi/MEKi,
                      42 (29 $\%)$ BRAFi monotherapy, and 9 (6 $\%)$ MEKi
                      monotherapy. The ORR was 35 $\%$ and higher in V600-nonE/K
                      (45 $\%)$ than non-V600 melanomas (26 $\%,$ p = 0.025).
                      Median duration of response was similar, with 8.2 months
                      (range 2.9-53.1 +) for V600-nonE/K and 7.4 months (range
                      0.8-73.8 +) for non-V600. Combination therapy achieved best
                      results in both groups, however, differences between
                      V600-nonE/K and non-V600mutation were only found in ORR (51
                      $\%$ vs. 33 $\%,$ p = 0,11) and median PFS (6.5 vs. 3.2
                      months, p = 0.01). Patients with the longest PFS (> 50
                      months) had V600D/R, $V600_K601D/E/N$ or K601E/N-,
                      L597V/S/R/Q/P/K- mutations. OS was similar in both groups
                      (16.1 vs. 11.7 months, p = 0.96). Of note, in non-V600
                      melanomas MEKi monotherapy revealed similar response rates
                      as combination treatment (ORR 33 $\%,$ PFS 3 months);
                      however, median OS was shorter (6.6 months, p = 0.02).This
                      updated analysis reinforces the benefit of BRAFi/MEKi
                      therapy in rare BRAF mutations. A database for ongoing data
                      collection was developed and is available at
                      https://www.klinikum.uni-heidelberg.de/en/hautklinik-zentrum/hauttumorzentrum/forschung/datenbank-seltene-braf-mutationen.},
      keywords     = {BRAFi (Other) / MEKi (Other) / Melanoma (Other) / Non-V600
                      (Other) / Rare BRAF mutation (Other) / Targeted therapy
                      (Other) / V600 (Other)},
      cin          = {ED01 / C060},
      ddc          = {610},
      cid          = {I:(DE-He78)ED01-20160331 / I:(DE-He78)C060-20160331},
      pnm          = {313 - Krebsrisikofaktoren und Prävention (POF4-313)},
      pid          = {G:(DE-HGF)POF4-313},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:40850313},
      doi          = {10.1016/j.ejca.2025.115703},
      url          = {https://inrepo02.dkfz.de/record/304097},
}