- Respondek, Gesine;
- Grimm, Max-Joseph;
- Piot, Ines;
- Arzberger, Thomas;
- Compta, Yaroslau;
- Englund, Elisabet;
- Ferguson, Leslie W;
- Gelpi, Ellen;
- Roeber, Sigrun;
- Giese, Armin;
- Grossman, Murray;
- Irwin, David J;
- Meissner, Wassilios G;
- Nilsson, Christer;
- Pantelyat, Alexander;
- Rajput, Alex;
- van Swieten, John C;
- Troakes, Claire;
- Höglinger, Günter U;
- Movement Disorder Society-Endorsed Progressive Supranuclear Palsy Study Group
Background
The Movement Disorder Society criteria for progressive supranuclear palsy introduced the category "probable 4-repeat (4R)-tauopathy" for joint clinical diagnosis of progressive supranuclear palsy and corticobasal degeneration.Objectives
To validate the accuracy of these clinical criteria for "probable 4R-tauopathy" to predict underlying 4R-tauopathy pathology.Methods
Diagnostic accuracy for 4R-tauopathies according to the established criteria was estimated retrospectively in autopsy-confirmed patients with progressive supranuclear palsy and corticobasal degeneration (grouped as 4R-tauopathies), and Parkinson's disease, multiple system atrophy, and frontotemporal lobar degeneration (grouped as non-4R-tauopathies).Results
We identified 250 cases with progressive supranuclear palsy (N = 195) and corticobasal degeneration (N = 55) and with and non-4R-tauopathies (N = 161). Sensitivity and specificity of "probable 4R-tauopathy" was 10% and 99% in the first year and 59% and 88% at final record.Conclusions
The new diagnostic category "probable 4R-tauopathy" showed high specificity and may be suitable for the recruitment of patients with progressive supranuclear palsy and corticobasal degeneration into therapeutic trials targeting 4R-tauopathy. The low sensitivity underpins the need for diagnostic biomarkers. © 2019 International Parkinson and Movement Disorder Society.