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@ARTICLE{vanTilburg:170170,
      author       = {C. M. van Tilburg and E. Pfaff and K. W. Pajtler$^*$ and K.
                      P. S. Langenberg and P. Fiesel and B. C. Jones and G. P.
                      Balasubramanian and S. Stark and P. D. Johann and M.
                      Blattner-Johnson and K. Schramm and N. Dikow and S. Hirsch
                      and C. Sutter and K. Grund and A. von Stackelberg and A. E.
                      Kulozik and A. Lissat and A. Borkhardt and R. Meisel and D.
                      Reinhardt and J.-H. Klusmann and G. Fleischhack and S.
                      Tippelt and D. von Schweinitz and I. Schmid and C. M. Kramm
                      and A. O. von Bueren and G. Calaminus and P. Vorwerk and N.
                      Graf and F. Westermann and M. Fischer and A. Eggert and B.
                      Burkhardt and W. Wossmann and M. Nathrath and S.
                      Hecker-Nolting and M. C. Fruhwald and D. T. Schneider and I.
                      B. Brecht and P. Ketteler and S. Fulda and E. Koscielniak
                      and M. T. Meister and M. Scheer and S. Hettmer and M. Schwab
                      and R. Tremmel and I. Ora and C. Hutter and N. U. Gerber and
                      O. Lohi and B. Kazanowska and A. Kattamis and M. Filippidou
                      and B. Goemans and C. M. Zwaan and T. Milde and N. Jäger
                      and S. Wolf$^*$ and D. Reuss$^*$ and F. Sahm$^*$ and A. von
                      Deimling$^*$ and U. Dirksen and A. Freitag and R. Witt and
                      P. Lichter$^*$ and A. Kopp-Schneider$^*$ and D. T. W.
                      Jones$^*$ and J. J. Molenaar and D. Capper and S. Pfister
                      and O. Witt$^*$},
      title        = {{T}he pediatric precision oncology {INFORM} registry:
                      clinical outcome and benefit for patients with very
                      high-evidence targets.},
      journal      = {Cancer discovery},
      volume       = {11},
      number       = {11},
      issn         = {2159-8290},
      address      = {Philadelphia, Pa.},
      reportid     = {DKFZ-2021-01794},
      pages        = {2764-2779},
      year         = {2021},
      note         = {#LA:B310# / 2021 Nov;11(11):2764-2779},
      abstract     = {INFORM is a prospective, multi-national registry gathering
                      clinical and molecular data of relapsed, progressive or
                      high-risk pediatric cancer patients. This report describes
                      long-term follow-up of 519 patients in whom molecular
                      alterations were evaluated according to a pre-defined
                      7-scale target prioritization algorithm. Mean turnaround
                      time from sample receipt to report was 25.4 days. The
                      highest target priority level was observed in 42 patients
                      $(8.1\%).$ Of these, twenty patients received matched
                      targeted treatment with a median PFS of 204 days $(95\%$ CI
                      99 - N.A.), compared with 117 days $(95\%$ CI 106 - 143;
                      P=0.011) in all other patients. The respective molecular
                      targets were shown to be predictive for matched treatment
                      response and not prognostic surrogates for improved outcome.
                      Hereditary cancer predisposition syndromes were identified
                      in $7.5\%$ of patients, half of which were newly identified
                      through the study. Integrated molecular analyses resulted in
                      a change or refinement of diagnoses in $8.2\%$ of cases.},
      cin          = {B062 / HD01 / B310 / B300 / W190 / B060 / C060 / B360},
      ddc          = {610},
      cid          = {I:(DE-He78)B062-20160331 / I:(DE-He78)HD01-20160331 /
                      I:(DE-He78)B310-20160331 / I:(DE-He78)B300-20160331 /
                      I:(DE-He78)W190-20160331 / I:(DE-He78)B060-20160331 /
                      I:(DE-He78)C060-20160331 / I:(DE-He78)B360-20160331},
      pnm          = {312 - Funktionelle und strukturelle Genomforschung
                      (POF4-312)},
      pid          = {G:(DE-HGF)POF4-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:34373263},
      doi          = {10.1158/2159-8290.CD-21-0094},
      url          = {https://inrepo02.dkfz.de/record/170170},
}