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@ARTICLE{Steinbgl:170092,
      author       = {M. Steinbügl and K. Nemes and P. Johann$^*$ and T.
                      Kröncke and S. Tüchert and M. J. G. da Costa and M.
                      Ebinger and U. Schüller and A. Sehested and P. Hauser and
                      H. Reinhard and D. Sumerauer and S. Hettmer and M. Jakob and
                      M. Hasselblatt and R. Siebert and O. Witt$^*$ and J. Gerss
                      and K. Kerl and M. C. Frühwald},
      title        = {{C}linical evidence for a biological effect of
                      epigenetically active decitabine in relapsed or progressive
                      rhabdoid tumors.},
      journal      = {Pediatric blood $\&$ cancer},
      volume       = {68},
      number       = {12},
      issn         = {1545-5017},
      address      = {New York, NY},
      publisher    = {Wiley},
      reportid     = {DKFZ-2021-01755},
      pages        = {e29267},
      year         = {2021},
      note         = {2021 Dec;68(12):e29267},
      abstract     = {Refined therapy has helped to improve survival rates in
                      rhabdoid tumors (RT). Prognosis for patients with
                      chemoresistant, recurrent, or progressive RT remains dismal.
                      Although decitabine, an epigenetically active agent, has
                      mainly been evaluated in the management of hematologic
                      malignancies in adults, safety in children has also been
                      demonstrated repeatedly.A retrospective series of patients
                      who received decitabine upon relapse or progression
                      following therapy according to the EU-RHAB regimen is
                      presented. Due to the retrospective nature of analyses,
                      response was defined as measurable regression of at least
                      one lesion on imaging. 850k methylation profiling was done
                      whenever tumor tissue was available.A total of 22 patients
                      with RT of any anatomical localization were included. Most
                      patients (19/22) presented with metastases. All received
                      low-dose decitabine with or preceding conventional
                      chemotherapy. Patients received a median of two (1-6)
                      courses of decitabine; $27.3\%$ (6/22) demonstrated a
                      radiological response. Molecular analyses revealed increased
                      methylation levels in tumors from responders. No excessive
                      toxicity was observed. Clinical benefits for responders
                      included eligibility for early phase trials or local
                      therapy. Responders showed prolonged time to progression and
                      overall survival. Due to small sample size, statistical
                      correction for survivorship bias demonstrated no significant
                      effect on survival for responders.Patients with RT
                      demonstrate promising signs of antitumor activity after
                      multiagent relapse therapy including decitabine. Analyses of
                      methylation data suggest a specific effect on an epigenetic
                      level. We propose to consider decitabine and other
                      epigenetic drugs as candidates for further clinical
                      investigations in RT.},
      keywords     = {ATRT (Other) / decitabine (Other) / malignant rhabdoid
                      tumor (Other) / relapsed and refractory rhabdoid tumors
                      (Other)},
      cin          = {B062 / B310},
      ddc          = {610},
      cid          = {I:(DE-He78)B062-20160331 / I:(DE-He78)B310-20160331},
      pnm          = {312 - Funktionelle und strukturelle Genomforschung
                      (POF4-312)},
      pid          = {G:(DE-HGF)POF4-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:34347371},
      doi          = {10.1002/pbc.29267},
      url          = {https://inrepo02.dkfz.de/record/170092},
}