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@ARTICLE{ObrechtSturm:271966,
      author       = {Obrecht-Sturm, Denise and Schömig, Lena and Mynarek,
                      Martin and Bison, Brigitte and Schwarz, Rudolf and Pietsch,
                      Torsten and Pfister, Stefan M and Sill, Martin and Sturm,
                      Dominik and Sahm, Felix and Kortmann, Rolf-Dieter and
                      Gerber, Nicolas U and von Bueren, André O and Fleischhack,
                      Gudrun and Schüller, Ulrich and Nussbaumer, Gunther and
                      Benesch, Martin and Rutkowski, Stefan},
      title        = {{T}reatment response as surrogate to predict risk for
                      disease progression in pediatric medulloblastoma with
                      persistent magnetic resonance imaging lesions after
                      first-line treatment.},
      journal      = {Neuro-Oncology},
      volume       = {26},
      number       = {9},
      issn         = {1522-8517},
      address      = {Oxford},
      publisher    = {Oxford Univ. Press},
      reportid     = {DZNE-2024-01108},
      pages        = {1712 - 1722},
      year         = {2024},
      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 magnetic resonance imaging (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, minor response [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 = .03; pptOS: 79.7 ± 5.9[MR/PR] vs.
                      55.5 ± 13.9[SD], P = .04). Furthermore, 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 = .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 = .71; 5-year pptOS: 76.3 ± $6.9\%$ vs. 69.8 ±
                      $9.7\%,$ P = .74). For the whole cohort, 5-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 = .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 the case of solitary
                      persistent medulloblastoma MRI lesions, additional therapy
                      was not beneficial. Therefore, treatment response,
                      extent/kind of residual lesions and further diagnostic
                      information need consideration for indication of additional
                      treatments for persisting lesions.},
      keywords     = {Humans / Medulloblastoma: diagnostic imaging /
                      Medulloblastoma: pathology / Male / Child / Female /
                      Magnetic Resonance Imaging: methods / Cerebellar Neoplasms:
                      diagnostic imaging / Cerebellar Neoplasms: pathology /
                      Disease Progression / Child, Preschool / Adolescent /
                      Follow-Up Studies / Prognosis / Retrospective Studies /
                      Survival Rate / Infant / Neoplasm, Residual: diagnostic
                      imaging / Neoplasm, Residual: pathology / MRI (Other) /
                      children (Other) / medulloblastoma (Other) / persistent
                      residual disease (Other)},
      cin          = {Brainbank (Bonn)},
      ddc          = {610},
      cid          = {I:(DE-2719)1011009},
      pnm          = {353 - Clinical and Health Care Research (POF4-353)},
      pid          = {G:(DE-HGF)POF4-353},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:38578306},
      pmc          = {pmc:PMC11376455},
      doi          = {10.1093/neuonc/noae071},
      url          = {https://pub.dzne.de/record/271966},
}