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@ARTICLE{Hglinger:139314,
      author       = {Höglinger, Günter U and Schöpe, Jakob and Stamelou,
                      Maria and Kassubek, Jan and Del Ser, Teodoro and Boxer, Adam
                      L and Wagenpfeil, Stefan and Huppertz, Hans-Jürgen and
                      Investigators, AL-108-231 and Investigators, Tauros MRI and
                      Group, Movement Disorder Society-Endorsed PSP Study},
      title        = {{L}ongitudinal magnetic resonance imaging in progressive
                      supranuclear palsy: {A} new combined score for clinical
                      trials.},
      journal      = {Movement disorders},
      volume       = {32},
      number       = {6},
      issn         = {0885-3185},
      address      = {New York, NY},
      publisher    = {Wiley},
      reportid     = {DZNE-2020-05636},
      pages        = {842-852},
      year         = {2017},
      abstract     = {Two recent, randomized, placebo-controlled phase II/III
                      trials (clinicaltrials.gov: NCT01110720, NCT01049399) of
                      davunetide and tideglusib in progressive supranuclear palsy
                      (PSP) generated prospective, 1-year longitudinal datasets of
                      high-resolution T1-weighted three-dimensional MRI.The
                      objective of this study was to develop a quantitative MRI
                      disease progression measurement for clinical trials.The
                      authors performed a fully automated quantitative MRI
                      analysis employing atlas-based volumetry and provide sample
                      size calculations based on data collected in 99 PSP patients
                      assigned to placebo in these trials. Based on individual
                      volumes of 44 brain compartments and structures at baseline
                      and 52 weeks of follow-up, means and standard deviations of
                      annualized percentage volume changes were used to estimate
                      standardized effect sizes and the required sample sizes per
                      group for future 2-armed, placebo-controlled therapeutic
                      trials.The highest standardized effect sizes were found for
                      midbrain, frontal lobes, and the third ventricle. Using the
                      annualized percentage volume change of these structures to
                      detect a $50\%$ change in the 1-year progression $(80\%$
                      power, significance level $5\%)$ required lower numbers of
                      patients per group (third ventricle, n = 32; midbrain, n =
                      37; frontal lobe, n = 43) than the best clinical scale (PSP
                      rating scale total score, n = 58). A combination of volume
                      changes in these 3 structures reduced the number of required
                      patients to only 20 and correlated best with the progression
                      in the clinical scales.We propose the 1-year change in the
                      volumes of third ventricle, midbrain, and frontal lobe as
                      combined imaging read-out for clinical trials in PSP that
                      require the least number of patients for detecting efficacy
                      to reduce brain atrophy. © 2017 International Parkinson and
                      Movement Disorder Society.},
      keywords     = {Aged / Clinical Trials as Topic: methods / Disease
                      Progression / Female / Frontal Lobe: diagnostic imaging /
                      Humans / Longitudinal Studies / Magnetic Resonance Imaging:
                      methods / Male / Mesencephalon: diagnostic imaging / Middle
                      Aged / Supranuclear Palsy, Progressive: diagnostic imaging /
                      Third Ventricle: diagnostic imaging},
      cin          = {AG Höglinger 1},
      ddc          = {610},
      cid          = {I:(DE-2719)1110002},
      pnm          = {344 - Clinical and Health Care Research (POF3-344)},
      pid          = {G:(DE-HGF)POF3-344},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:28436538},
      pmc          = {pmc:PMC5808453},
      doi          = {10.1002/mds.26973},
      url          = {https://pub.dzne.de/record/139314},
}