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@ARTICLE{Schubert:154279,
      author       = {N. A. Schubert and C. D. Lowery and G. Bergthold and J.
                      Koster and T. F. Eleveld and A. Rodríguez and D. T. W.
                      Jones$^*$ and G. Vassal and L. F. Stancato and S. M.
                      Pfister$^*$ and H. Heidelberg University Hospital and H. N.
                      Caron and J. J. Molenaar},
      othercontributors = {H. German Cancer Research Center and H. German Cancer
                          Research Center},
      title        = {{S}ystematic target actionability reviews of preclinical
                      proof-of-concept papers to match targeted drugs to
                      paediatric cancers.},
      journal      = {European journal of cancer},
      volume       = {130},
      issn         = {0959-8049},
      address      = {Amsterdam [u.a.]},
      publisher    = {Elsevier},
      reportid     = {DKFZ-2020-00706},
      pages        = {168 - 181},
      year         = {2020},
      abstract     = {Children with cancer are in urgent need of new therapies,
                      as approximately $25\%$ of patients experience a relapse and
                      $20\%$ succumb to their disease. Moreover, the majority of
                      survivors suffer from clinically relevant health problems.
                      Repurposing of targeted agents developed for adult
                      indications could provide novel therapeutic options for
                      paediatric cancer patients. To prioritise targeted drugs for
                      paediatric clinical development, we applied a systematic
                      review methodology to develop a Target Actionability Review
                      (TAR) strategy. These TARs assess the strength and
                      completeness of published preclinical proof-of-concept (PoC)
                      data by structured critical appraisal of and summarising the
                      available scientific literature for a specific target
                      (pathway) and the associated drugs in paediatric tumours.A
                      sensitive literature search in PubMed was performed and
                      relevant papers were identified. For each paper, the
                      individual experimental findings were extracted, marked for
                      paediatric tumour type and categorised into nine separate
                      PoC data modules. Each experimental finding was scored for
                      experimental outcome and quality independently by two
                      reviewers; discrepancies were assessed by a third reviewer
                      and resolved by adjudication. Scores corresponding to one
                      PoC module were merged for each tumour type and visualised
                      in a heat map matrix in the publicly available R2 data
                      portal [r2.amc.nl].To test our TAR methodology, we conducted
                      a pilot study on MDM2 and TP53. The heat map generated from
                      analysis of 161 publications provides a rationale to support
                      drug development in specific paediatric solid and brain
                      tumour types. Furthermore, our review highlights tumour
                      types where preclinical data are incomplete or lacking and
                      for which additional preclinical testing is advisable.},
      subtyp        = {Review Article},
      cin          = {B360 / B062},
      ddc          = {610},
      cid          = {I:(DE-He78)B360-20160331 / I:(DE-He78)B062-20160331},
      pnm          = {312 - Functional and structural genomics (POF3-312)},
      pid          = {G:(DE-HGF)POF3-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:32224415},
      doi          = {10.1016/j.ejca.2020.01.027},
      url          = {https://inrepo02.dkfz.de/record/154279},
}