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@ARTICLE{Weidenbusch:136976,
      author       = {B. Weidenbusch and G. H. S. Richter and M. S. Kesper and M.
                      Guggemoos and K. Gall and C. Prexler and I. Kazantsev and A.
                      Sipol and L. Lindner and M. Nathrath and O. Witt$^*$ and K.
                      Specht and F. Beitinger and C. Knebel and S. Hosie and R.
                      von Eisenhardt-Rothe and W. Weichert and I. T. Luettichau
                      and S. Burdach},
      title        = {{T}ranscriptome based individualized therapy of refractory
                      pediatric sarcomas: feasibility, tolerability and efficacy.},
      journal      = {OncoTarget},
      volume       = {9},
      number       = {29},
      issn         = {1949-2553},
      address      = {[S.l.]},
      publisher    = {Impact Journals LLC},
      reportid     = {DKFZ-2018-01404},
      pages        = {20747-20760},
      year         = {2018},
      abstract     = {Survival rates of pediatric sarcoma patients stagnated
                      during the last two decades, especially in adolescents and
                      young adults (AYAs). Targeted therapies offer new options in
                      refractory cases. Gene expression profiling provides a
                      robust method to characterize the transcriptome of each
                      patient's tumor and guide the choice of therapy. Twenty
                      patients with refractory pediatric sarcomas (age 8-35 years)
                      were assessed with array profiling: ten had Ewing sarcoma,
                      five osteosarcoma, and five soft tissue sarcoma.
                      Overexpressed genes and deregulated pathways were identified
                      as actionable targets and an individualized combination of
                      targeted therapies was recommended. Disease status,
                      survival, adverse events (AEs), and quality of life (QOL)
                      were assessed in patients receiving targeted therapy (TT)
                      and compared to patients without targeted therapy (non TT).
                      Actionable targets were identified in all analyzed biopsies.
                      Targeted therapy was administered in nine patients, while
                      eleven received no targeted therapy. No significant
                      difference in risk factors between these two groups was
                      detected. Overall survival (OS) and progression free
                      survival (PFS) were significantly higher in the TT group
                      (OS: P=0.0014, PFS: P=0.0011). Median OS was 8.83 versus
                      4.93 months and median PFS was 6.17 versus 1.6 months in TT
                      versus non TT group, respectively. QOL did not differ at
                      baseline as well as at four week intervals between the two
                      groups. TT patients had less grade 1 AEs (P=0.009). The
                      frequency of grade 2-4 AEs did not differ. Overall,
                      expression based targeted therapy is a feasible and likely
                      beneficial approach in patients with refractory pediatric
                      sarcomas that warrants further study.},
      cin          = {G340},
      ddc          = {610},
      cid          = {I:(DE-He78)G340-20160331},
      pnm          = {317 - Translational cancer research (POF3-317)},
      pid          = {G:(DE-HGF)POF3-317},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:29755686},
      pmc          = {pmc:PMC5945512},
      doi          = {10.18632/oncotarget.25087},
      url          = {https://inrepo02.dkfz.de/record/136976},
}