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@ARTICLE{Schmidt:278341,
      author       = {T. Schmidt$^*$ and S. Agkatsev$^*$ and J. Feldheim$^*$ and
                      C. Oster$^*$ and T. Blau$^*$ and U. Sure$^*$ and K.
                      Keyvani$^*$ and C. Kleinschnitz$^*$ and M. Stuschke$^*$ and
                      K. Herrmann$^*$ and C. Deuschl$^*$ and B. Scheffler$^*$ and
                      S. Kebir$^*$ and M. Glas$^*$ and L. Lazaridis$^*$},
      title        = {{F}easibility and tolerability of trofosfamide and
                      etoposide in progressive glioblastoma.},
      journal      = {Neuro-oncology advances},
      volume       = {5},
      number       = {1},
      issn         = {2632-2498},
      address      = {Oxford},
      publisher    = {Oxford University Press},
      reportid     = {DKFZ-2023-01598},
      pages        = {vdad090},
      year         = {2023},
      abstract     = {Standard of care treatment options at glioblastoma relapse
                      are still not well defined. Few studies indicate that the
                      combination of trofosfamide plus etoposide may be feasible
                      in pediatric glioblastoma patients. In this retrospective
                      analysis, we determined tolerability and feasibility of
                      combined trofosfamide plus etoposide treatment at disease
                      recurrence of adult glioblastoma patients.We collected
                      clinicopathological data from adult progressive glioblastoma
                      patients treated with the combination of trofosfamide and
                      etoposide for more than four weeks (one course). A cohort of
                      patients receiving empiric treatment at the investigators'
                      discretion balanced for tumor entity and canonical
                      prognostic factors served as control.A total of n = 22
                      progressive glioblastoma patients were eligible for this
                      analysis. Median progression-free survival (3.1 vs 2.3
                      months, HR: 1.961, $95\%$ CI: 0.9724-3.9560, P = .0274) and
                      median overall survival (9.0 vs 5.7 months, HR: 4.687,
                      $95\%$ CI: 2.034-10.800, P = .0003) were significantly
                      prolonged compared to the control cohort (n = 17). In a
                      multivariable Cox regression analysis, treatment with
                      trofosfamide plus etoposide emerged as a significant
                      prognostic marker regarding progression-free and overall
                      survival. We observed high-grade adverse events in n = 16/22
                      $(73\%)$ patients with hematotoxicity comprising the
                      majority of adverse events (n = 15/16, $94\%).$ Lymphopenia
                      was by far the most commonly observed hematotoxic adverse
                      event (n = 11/15, $73\%).This$ study provides first
                      indication that the combination of trofosfamide plus
                      etoposide is safe in adult glioblastoma patients. The
                      observed survival outcomes might suggest potential
                      beneficial effects. Our data provide a reasonable rationale
                      for follow-up of a larger cohort in a prospective trial.},
      keywords     = {etoposide (Other) / glioblastoma (Other) / progressive
                      glioblastoma (Other) / trofosfamide (Other)},
      cin          = {ED01},
      ddc          = {610},
      cid          = {I:(DE-He78)ED01-20160331},
      pnm          = {899 - ohne Topic (POF4-899)},
      pid          = {G:(DE-HGF)POF4-899},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:37547266},
      pmc          = {pmc:PMC10403750},
      doi          = {10.1093/noajnl/vdad090},
      url          = {https://inrepo02.dkfz.de/record/278341},
}