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@ARTICLE{Edelmann:295924,
      author       = {D. Edelmann$^*$ and T. Terzer$^*$ and P. Horak$^*$ and R.
                      Schlenk and A. Benner$^*$},
      title        = {{T}he {P}rogression-{F}ree-{S}urvival {R}atio in
                      {M}olecularly {A}ided {T}umor {T}rials: {A} {C}ritical
                      {E}xamination of {C}urrent {P}ractice and {S}uggestions for
                      {A}lternative {M}ethods.},
      journal      = {Biometrical journal},
      volume       = {67},
      number       = {1},
      issn         = {0323-3847},
      address      = {Berlin},
      publisher    = {Wiley-VCH},
      reportid     = {DKFZ-2024-02737},
      pages        = {e70028},
      year         = {2025},
      note         = {#EA:C060#LA:C060#},
      abstract     = {The progression-free-survival ratio is a popular endpoint
                      in oncology trials, which is frequently applied to evaluate
                      the efficacy of molecularly targeted treatments in
                      late-stage patients. Using elementary calculations and
                      simulations, numerous shortcomings of the current
                      methodology are pointed out. As a remedy to these
                      shortcomings, an alternative methodology is proposed, using
                      a marginal Cox model or a marginal accelerated failure time
                      model for clustered time-to-event data. Using comprehensive
                      simulations, it is shown that this methodology outperforms
                      existing methods in settings where the intrapatient
                      correlation is low to moderate. The performance of the model
                      is further demonstrated in a real data example from a
                      molecularly aided tumor trial. Sample size considerations
                      are discussed.},
      keywords     = {Humans / Biometry: methods / Neoplasms: mortality /
                      Clinical Trials as Topic: methods / Progression-Free
                      Survival / Molecular Targeted Therapy: methods / Models,
                      Statistical / Proportional Hazards Models / Weibull
                      distribution (Other) / accelerated failure time model
                      (Other) / growth modulation index (Other) / marginal model
                      (Other) / paired time‐to‐event data (Other) /
                      progression‐free‐survival ratio (Other)},
      cin          = {C060 / B340},
      ddc          = {570},
      cid          = {I:(DE-He78)C060-20160331 / I:(DE-He78)B340-20160331},
      pnm          = {313 - Krebsrisikofaktoren und Prävention (POF4-313)},
      pid          = {G:(DE-HGF)POF4-313},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:39692541},
      doi          = {10.1002/bimj.70028},
      url          = {https://inrepo02.dkfz.de/record/295924},
}