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@ARTICLE{Liebers:284403,
      author       = {N. Liebers$^*$ and P.-M. Bruch and T. Terzer$^*$ and M.
                      Hernandez-Hernandez and N. Paramasivam and D. Fitzgerald and
                      H. Altmann and T. Roider and C. Kolb and M. Knoll and A.
                      Lenze and U. Platzbecker and C. Röllig and C. Baldus and H.
                      Serve and M. Bornhäuser and D. Hübschmann$^*$ and C.
                      Müller-Tidow$^*$ and F. Stölzel and W. Huber and A.
                      Benner$^*$ and T. Zenz and J. Lu and S. Dietrich$^*$},
      title        = {{E}x vivo drug response profiling for response and outcome
                      prediction in hematologic malignancies: the prospective
                      non-interventional {SMART}rial.},
      journal      = {Nature cancer},
      volume       = {4},
      number       = {12},
      issn         = {2662-1347},
      address      = {London},
      publisher    = {Nature Research},
      reportid     = {DKFZ-2023-01992},
      pages        = {1648-1659},
      year         = {2023},
      note         = {2023 Dec;4(12):1648-1659 / #EA:B340#LA:B340#},
      abstract     = {Ex vivo drug response profiling is a powerful tool to study
                      genotype-drug response associations and is being explored as
                      a tool set for precision medicine in cancer. Here we
                      conducted a prospective non-interventional trial to
                      investigate feasibility of ex vivo drug response profiling
                      for treatment guidance in hematologic malignancies
                      (SMARTrial, NCT03488641 ). The primary endpoint to provide
                      drug response profiling reports within 7 d was met in $91\%$
                      of all study participants (N = 80). Secondary endpoint
                      analysis revealed that ex vivo resistance to
                      chemotherapeutic drugs predicted chemotherapy treatment
                      failure in vivo. We confirmed the predictive value of ex
                      vivo response to chemotherapy in a validation cohort of 95
                      individuals with acute myeloid leukemia treated with
                      daunorubicin and cytarabine. Ex vivo drug response profiles
                      improved ELN-22 risk stratification in individuals with
                      adverse risk. We conclude that ex vivo drug response
                      profiling is clinically feasible and has the potential to
                      predict chemotherapy response in individuals with
                      hematologic malignancies beyond clinically established
                      genetic markers.},
      cin          = {B340 / C060 / HD01},
      ddc          = {610},
      cid          = {I:(DE-He78)B340-20160331 / I:(DE-He78)C060-20160331 /
                      I:(DE-He78)HD01-20160331},
      pnm          = {312 - Funktionelle und strukturelle Genomforschung
                      (POF4-312)},
      pid          = {G:(DE-HGF)POF4-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:37783805},
      doi          = {10.1038/s43018-023-00645-5},
      url          = {https://inrepo02.dkfz.de/record/284403},
}