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@ARTICLE{ObrechtSturm:289304,
      author       = {D. Obrecht-Sturm and L. Schömig and M. Mynarek and B.
                      Bison and R. Schwarz and T. Pietsch and S. Pfister$^*$ and
                      M. Sill$^*$ and D. Sturm$^*$ and F. Sahm$^*$ and R.-D.
                      Kortmann and N. U. Gerber and A. O. von Bueren and G.
                      Fleischhack and U. Schüller and G. Nussbaumer and M.
                      Benesch and S. Rutkowski},
      title        = {{T}reatment response as surrogate to predict risk for
                      disease progression in pediatric medulloblastoma with
                      persistent {MRI} lesions after first-line treatment.},
      journal      = {Neuro-Oncology},
      volume       = {26},
      number       = {9},
      issn         = {1522-8517},
      address      = {Oxford},
      publisher    = {Oxford Univ. Press},
      reportid     = {DKFZ-2024-00699},
      pages        = {1712-1722},
      year         = {2024},
      note         = {2024 Sep 5;26(9):1712-1722},
      abstract     = {This study aims at clarifying the impact of persistent
                      residual lesions following first-line treatment for
                      pediatric medulloblastoma.Data on 84 pediatric patients with
                      medulloblastoma and persistent residual lesions on centrally
                      reviewed MRI at the end of first-line therapy were
                      analyzed.Twenty patients $(23.8\%)$ had residual lesions in
                      the tumor bed (R+/M0), 51 $(60.7\%)$ had distant lesions
                      (R0/M+) and 13 $(15.5\%)$ had both (R+/M+). Overall response
                      to first-line therapy was minor or partial $(≥25\%$
                      reduction, MR/PR) for 64 $(76.2\%)$ and stable disease (SD)
                      for 20 patients $(23.8\%).$ Five-year post-primary-treatment
                      progression-free (pptPFS) and overall survival (pptOS) were
                      superior after MR/PR (pptPFS: $62.5±7.0\%[MR/PR]$ vs.
                      $35.9±12.8\%[SD],$ p=0.03; pptOS: 79.7±5.9[MR/PR] vs.
                      55.5±13.9[SD], p=0.04). Further, R+/M+ was associated with
                      a higher risk for progression (5-year pptPFS:
                      $22.9±17.9\%[R+,M+]$ vs. $72.4±12.0\%[R+,M0];$ p=0.03).
                      Watch-and-wait was pursued in 58 patients, while n=26
                      received additional treatments (chemotherapy only, n=19;
                      surgery only, n=2; combined, n=3; valproic acid, n=2), and
                      their outcomes were not superior to watch-and-wait (5-year
                      pptPFS: $58.5±7.7\%$ vs. $51.6±10.7\%$ p=0.71; 5-year
                      pptOS: $76.3±6.9\%$ vs. $69.8±9.7\%,$ p=0.74). For the
                      whole cohort, five-year pptPFS by molecular subgroup (58
                      cases) were WNT: $100\%,$ SHH: $50.0±35.4\%,$ Group-4,
                      52.5±10.5, Group-3 $54.2±13.8\%;$ (p=0.08).Overall
                      response and extent of lesions can function as surrogate
                      parameters to predict outcomes in pediatric MB patients with
                      persistent lesions after first-line therapy. Especially in
                      case of solitary persistent medulloblastoma MRI lesion,
                      additional therapy was not beneficial. Therefore, treatment
                      response, extent/kind of residual lesions and further
                      diagnostic information needs consideration for indication of
                      additional treatments for persisting lesions.},
      keywords     = {MRI (Other) / children (Other) / medulloblastoma (Other) /
                      persistent residual disease (Other)},
      cin          = {B062 / HD01 / B360 / B300},
      ddc          = {610},
      cid          = {I:(DE-He78)B062-20160331 / I:(DE-He78)HD01-20160331 /
                      I:(DE-He78)B360-20160331 / I:(DE-He78)B300-20160331},
      pnm          = {312 - Funktionelle und strukturelle Genomforschung
                      (POF4-312)},
      pid          = {G:(DE-HGF)POF4-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:38578306},
      doi          = {10.1093/neuonc/noae071},
      url          = {https://inrepo02.dkfz.de/record/289304},
}