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@ARTICLE{Benesch:143462,
      author       = {M. Benesch and M. Mynarek and H. Witt$^*$ and M.
                      Warmuth-Metz and T. Pietsch and B. Bison and S. Pfister$^*$
                      and K. Pajtler$^*$ and M. Kool$^*$ and U. Schüller and K.
                      Pietschmann and B.-O. Juhnke and S. Tippelt and G.
                      Fleischhack and I. Schmid and C. M. Kramm and P. Vorwerk and
                      A. Beilken and C. F. Classen and P. Hernáiz Driever and G.
                      Kropshofer and T. Imschweiler and A. Lemmer and R.-D.
                      Kortmann and S. Rutkowski and K. von Hoff},
      title        = {{N}ewly {D}iagnosed {M}etastatic {I}ntracranial
                      {E}pendymoma in {C}hildren: {F}requency, {M}olecular
                      {C}haracteristics, {T}reatment, and {O}utcome in the
                      {P}rospective {HIT} {S}eries.},
      journal      = {The oncologist},
      volume       = {24},
      number       = {9},
      issn         = {1549-490X},
      address      = {Hoboken, NJ},
      publisher    = {Wiley},
      reportid     = {DKFZ-2019-01050},
      pages        = {E921-E929},
      year         = {2019},
      abstract     = {Data on frequency, clinical presentation, and outcome of
                      primary metastatic intracranial ependymoma in children are
                      scarce.Prospective data on patients younger than 21 years
                      with metastatic intracranial ependymoma at first diagnosis,
                      registered from 2001 to 2014 in the HIT-2000 trial and the
                      HIT-2000 Interim Registry, were analyzed.Of 453 registered
                      patients with intracranial ependymoma and central
                      neuropathology review, initial staging included spinal
                      magnetic resonance imaging in all patients and lumbar
                      cerebrospinal fluid (CSF) analysis in 402 patients. Ten
                      patients $(2.2\%)$ had metastatic disease, including three
                      with microscopic CSF positivity only (M1 metastasis stage,
                      $0.7\%$ of patients with CSF staging). Location of the
                      primary tumor was supratentorial in four patients (all
                      supratentorial RELA-fused ependymoma [ST-EPN-RELA]) and
                      within the posterior fossa in five patients (posterior fossa
                      ependymoma type A [PF-EPN-A], n = 4; posterior fossa
                      ependymoma not further classifiable, n = 1), and
                      multifocal in one patient.All four patients with ST-EPN-RELA
                      were alive in first or second complete remission (CR)
                      7.5-12.3 years after diagnosis. All four patients with
                      macroscopic metastases of posterior fossa or multifocal
                      ependymoma died. Three patients with initial M1 stage
                      (ST-EPN-RELA, n = 1; PF-EPN-A, n = 2) received
                      chemotherapy and local irradiation and were alive in second
                      or third CR 3.0-9.7 years after diagnosis.
                      Progression-free and overall survival of the entire cohort
                      at 5 years was $13\%$ $(±6\%),$ and $58\%$ $(±16\%),$
                      respectively.Primary metastatic disease is rare in children
                      with intracranial ependymoma. Prognosis may depend on
                      molecular subgroup and extent of dissemination, and
                      relevance of CSF analysis for initial staging remains to be
                      clarified.Childhood ependymoma presenting with metastasis at
                      first diagnosis is very rare with a frequency of $2.4\%$ in
                      this population-based, well-characterized cohort. Detection
                      of microscopic metastases in the cerebrospinal fluid was
                      extremely rare, and impact on prognosis and respective
                      treatment decision on irradiation field remains unclear.
                      Initial metastatic presentation occurs in both
                      supratentorial RELA-fused ependymoma and posterior fossa
                      ependymoma. Prognosis may differ according to extent of
                      metastasis and biological subgroup, with poor prognosis in
                      diffusely spread metastatic posterior fossa ependymoma even
                      after combination therapy with both intensive chemotherapy
                      and craniospinal irradiation, which may help to guide
                      individual therapeutic decisions for future patients.},
      cin          = {B062 / L101},
      ddc          = {610},
      cid          = {I:(DE-He78)B062-20160331 / I:(DE-He78)L101-20160331},
      pnm          = {312 - Functional and structural genomics (POF3-312)},
      pid          = {G:(DE-HGF)POF3-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:30850560},
      doi          = {10.1634/theoncologist.2018-0489},
      url          = {https://inrepo02.dkfz.de/record/143462},
}