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@ARTICLE{Gajjar:166689,
      author       = {A. Gajjar and G. W. Robinson and K. S. Smith and T. Lin and
                      T. E. Merchant and M. Chintagumpala and A. Mahajan and J. Su
                      and E. Bouffet and U. Bartels and T. Schechter and T.
                      Hassall and T. Robertson and W. Nicholls and S. Gururangan
                      and K. Schroeder and M. Sullivan and G. Wheeler and J. R.
                      Hansford and S. J. Kellie and G. McCowage and R. Cohn and M.
                      J. Fisher and M. J. Krasin and C. F. Stewart and A.
                      Broniscer and I. Buchhalter$^*$ and R. G. Tatevossian and B.
                      A. Orr and G. Neale and P. Klimo and F. Boop and A.
                      Srinivasan and S. M. Pfister$^*$ and R. J. Gilbertson and A.
                      Onar-Thomas and D. W. Ellison and P. A. Northcott},
      title        = {{O}utcomes by {C}linical and {M}olecular {F}eatures in
                      {C}hildren {W}ith {M}edulloblastoma {T}reated {W}ith
                      {R}isk-{A}dapted {T}herapy: {R}esults of an {I}nternational
                      {P}hase {III} {T}rial ({SJMB}03).},
      journal      = {Journal of clinical oncology},
      volume       = {39},
      number       = {7},
      issn         = {1527-7755},
      address      = {Alexandria, Va.},
      publisher    = {American Society of Clinical Oncology},
      reportid     = {DKFZ-2021-00042},
      pages        = {822-835},
      year         = {2021},
      note         = {2021 Mar 1;39(7):822-835},
      abstract     = {SJMB03 (ClinicalTrials.gov identifier: NCT00085202) was a
                      phase III risk-adapted trial that aimed to determine the
                      frequency and clinical significance of biological variants
                      and genetic alterations in medulloblastoma.Patients 3-21
                      years old were stratified into average-risk and high-risk
                      treatment groups based on metastatic status and extent of
                      resection. Medulloblastomas were molecularly classified into
                      subgroups (Wingless [WNT], Sonic Hedgehog [SHH], group 3,
                      and group 4) and subtypes based on DNA methylation profiles
                      and overlaid with gene mutations from next-generation
                      sequencing. Coprimary study end points were (1) to assess
                      the relationship between ERBB2 protein expression in tumors
                      and progression-free survival (PFS), and (2) to estimate the
                      frequency of mutations associated with WNT and SHH tumors.
                      Clinical and molecular risk factors were evaluated, and the
                      most robust were used to model new risk-classification
                      categories.Three hundred thirty eligible patients with
                      medulloblastoma were enrolled. Five-year PFS was $83.2\%$
                      $(95\%$ CI, 78.4 to 88.2) for average-risk patients (n =
                      227) and $58.7\%$ $(95\%$ CI, 49.8 to 69.1) for high-risk
                      patients (n = 103). No association was found between ERBB2
                      status and PFS in the overall cohort (P = .74) or when
                      patients were stratified by clinical risk (P = .71).
                      Mutations in CTNNB1 $(96\%),$ DDX3X $(37\%),$ and SMARCA4
                      $(24\%)$ were most common in WNT tumors and PTCH1 $(38\%),$
                      TP53 $(21\%),$ and DDX3X $(19\%)$ in SHH tumors. Methylome
                      profiling classified 53 WNT $(17.4\%),$ 48 SHH $(15.7\%),$
                      65 group 3 $(21.3\%),$ and 139 group 4 $(45.6\%)$ tumors. A
                      comprehensive clinicomolecular risk factor analysis
                      identified three low-risk groups (WNT, low-risk SHH, and
                      low-risk combined groups 3 and 4) with excellent (5-year PFS
                      > $90\%)$ and two very high-risk groups (high-risk SHH and
                      high-risk combined groups 3 and 4) with poor survival
                      (5-year PFS < $60\%).These$ results establish a new risk
                      stratification for future medulloblastoma trials.},
      cin          = {W610 / B062 / HD01},
      ddc          = {610},
      cid          = {I:(DE-He78)W610-20160331 / I:(DE-He78)B062-20160331 /
                      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:33405951},
      doi          = {10.1200/JCO.20.01372},
      url          = {https://inrepo02.dkfz.de/record/166689},
}