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@ARTICLE{Hau:169962,
      author       = {P. Hau and D. Frappaz and E. Hovey and M. G. McCabe and K.
                      W. Pajtler$^*$ and B. Wiestler and C. Seidel and S. E. Combs
                      and L. Dirven and M. Klein and A. Anazodo and E. Hattingen
                      and S. Hofer and S. M. Pfister$^*$ and C. Zimmer and R.-D.
                      Kortmann and M.-P. Sunyach and R. Tanguy and R. Effeney and
                      A. von Deimling$^*$ and F. Sahm$^*$ and S. Rutkowski$^*$ and
                      A. S. Berghoff and E. Franceschi and E. Pineda and D. Beier
                      and E. Peeters and T. Gorlia and M. Vanlancker and J. E. C.
                      Bromberg and J. Gautier and D. S. Ziegler and M. Preusser
                      and W. Wick$^*$ and M. Weller},
      title        = {{D}evelopment of {R}andomized {T}rials in {A}dults with
                      {M}edulloblastoma-{T}he {E}xample of {EORTC}
                      1634-{BTG}/{NOA}-23.},
      journal      = {Cancers},
      volume       = {13},
      number       = {14},
      issn         = {2072-6694},
      address      = {Basel},
      publisher    = {MDPI},
      reportid     = {DKFZ-2021-01662},
      pages        = {3451},
      year         = {2021},
      abstract     = {Medulloblastoma is a rare brain malignancy. Patients after
                      puberty are rare and bear an intermediate prognosis.
                      Standard treatment consists of maximal resection plus
                      radio-chemotherapy. Treatment toxicity is high and produces
                      disabling long-term side effects. The sonic hedgehog (SHH)
                      subgroup is highly overrepresented in the post-pubertal and
                      adult population and can be targeted by smoothened (SMO)
                      inhibitors. No practice-changing prospective randomized data
                      have been generated in adults. The EORTC 1634-BTG/NOA-23
                      trial will randomize patients between standard-dose vs.
                      reduced-dosed craniospinal radiotherapy and SHH-subgroup
                      patients between the SMO inhibitor sonidegib (OdomzoTM, Sun
                      Pharmaceuticals Industries, Inc., New York, USA) in addition
                      to standard radio-chemotherapy vs. standard
                      radio-chemotherapy alone to improve outcomes in view of
                      decreased radiotherapy-related toxicity and increased
                      efficacy. We will further investigate tumor tissue, blood,
                      and cerebrospinal fluid as well as magnetic resonance
                      imaging and radiotherapy plans to generate information that
                      helps to further improve treatment outcomes. Given that
                      treatment side effects typically occur late, long-term
                      follow-up will monitor classic side effects of therapy, but
                      also health-related quality of life, cognition, social and
                      professional outcome, and reproduction and fertility. In
                      summary, we will generate unprecedented data that will be
                      translated into treatment changes in post-pubertal patients
                      with medulloblastoma and will help to design future clinical
                      trials.},
      subtyp        = {Review Article},
      keywords     = {adult (Other) / cerebrospinal fluid (Other) / chemotherapy
                      (Other) / clinical trial (Other) / magnetic resonance
                      imaging (Other) / medulloblastoma (Other) / radiotherapy
                      (Other) / randomized (Other) / subgrouping (Other) /
                      targeted therapy (Other)},
      cin          = {HD01 / B062 / B320},
      ddc          = {610},
      cid          = {I:(DE-He78)HD01-20160331 / I:(DE-He78)B062-20160331 /
                      I:(DE-He78)B320-20160331},
      pnm          = {312 - Funktionelle und strukturelle Genomforschung
                      (POF4-312)},
      pid          = {G:(DE-HGF)POF4-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:34298664},
      doi          = {10.3390/cancers13143451},
      url          = {https://inrepo02.dkfz.de/record/169962},
}