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@ARTICLE{Obrecht:178901,
      author       = {D. Obrecht and M. Mynarek and C. Hagel and R. Kwiecien and
                      M. Spohn and M. Bockmayr and B. Bison and S. M. Pfister$^*$
                      and D. T. W. Jones$^*$ and D. Sturm$^*$ and A. von
                      Deimling$^*$ and F. Sahm$^*$ and K. von Hoff and B.-O.
                      Juhnke and M. Benesch and N. U. Gerber and C. Friedrich and
                      A. O. von Bueren and R.-D. Kortmann and R. Schwarz and T.
                      Pietsch and G. Fleischhack and U. Schüller and S.
                      Rutkowski},
      title        = {{C}linical and molecular characterization of isolated {M}1
                      disease in pediatric medulloblastoma: experience from the
                      {G}erman {HIT}-{MED} studies.},
      journal      = {Journal of neuro-oncology},
      volume       = {157},
      number       = {1},
      issn         = {0167-594X},
      address      = {Dordrecht [u.a.]},
      publisher    = {Springer Science + Business Media B.V},
      reportid     = {DKFZ-2022-00332},
      pages        = {37-48},
      year         = {2022},
      note         = {2022 Mar;157(1):37-48},
      abstract     = {To evaluate the clinical impact of isolated spread of
                      medulloblastoma cells into cerebrospinal fluid without
                      additional macroscopic metastases (M1-only).The HIT-MED
                      database was searched for pediatric patients with M1-only
                      medulloblastoma diagnosed from 2000 to 2019. Corresponding
                      clinical and molecular data was evaluated. Treatment was
                      stratified by age and changed over time for older
                      patients.70 patients with centrally reviewed M1-only disease
                      were identified. Clinical data was available for all and
                      molecular data for 45/70 cases. $91\%$ were non-WNT/non-SHH
                      medulloblastoma (Grp3/4). 5-year PFS for 52 patients ≥
                      4 years was 59.4 (± 7.1) $\%,$ receiving either upfront
                      craniospinal irradiation (CSI) or SKK-sandwich chemotherapy
                      (CT). Outcomes did not differ between these strategies
                      (5-year PFS: CSI 61.7 ± $9.9\%,$ SKK-CT 56.7 ± $6.1\%).$
                      For patients < 4 years (n = 18), 5-year PFS was 50.0 (±
                      13.2) $\%.$ M1-persistence occurred exclusively using
                      postoperative CT and was a strong negative predictive factor
                      (pPFS/OS < 0.01). Patients with additional clinical or
                      molecular high-risk (HR) characteristics had worse outcomes
                      (5-year PFS 42.7 ± $10.6\%$ vs. 64.0 ± $7.0\%,$ p = 0.03).
                      In n = 22 patients ≥ 4 years with full molecular
                      information and without additional HR characteristics, risk
                      classification by molecular subtyping had an effect on
                      5-year PFS (HR 16.7 ± $15.2\%,$ SR 77.8 ± $13.9\%;$ p =
                      0.01).Our results confirm that M1-only is a high-risk
                      condition, and further underline the importance of CSF
                      staging. Specific risk stratification of affected patients
                      needs attention in future discussions for trials and
                      treatment recommendations. Future patients without
                      contraindications may benefit from upfront CSI by sparing
                      risks related to higher cumulative CT applied in sandwich
                      regimen.},
      keywords     = {Cerebrospinal fluid (Other) / Children (Other) /
                      Medulloblastoma (Other) / Metastases (Other) / Radiotherapy
                      (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:35190934},
      doi          = {10.1007/s11060-021-03913-5},
      url          = {https://inrepo02.dkfz.de/record/178901},
}