DescribePSP and ProPSP: German Multicenter Networks for Standardized Prospective Collection of Clinical Data, Imaging Data, and Biomaterials of Patients With Progressive Supranuclear Palsy.

Gesine Respondek,Günter U Höglinger

doi:10.3389/fneur.2021.644064

Abstract

Background: The German research networks DescribePSP and ProPSP prospectively collect comprehensive clinical data, imaging data and biomaterials of patients with a clinical diagnosis of progressive supranuclear palsy. Progressive supranuclear palsy is a rare, adult-onset, neurodegenerative disease with striking clinical heterogeneity. Since now, prospective natural history data are largely lacking. Clinical research into treatment strategies has been limited due to delay in clinical diagnosis and lack of natural history data on distinct clinical phenotypes.Methods: The DescribePSP network is organized by the German Center for Neurodegenerative Diseases. DescribePSP is embedded in a larger network with parallel cohorts of other neurodegenerative diseases and healthy controls. The DescribePSP network is directly linked to other Describe cohorts with other primary diagnoses of the neurodegenerative and vascular disease spectrums and also to an autopsy program for clinico-pathological correlation. The ProPSP network is organized by the German Parkinson and Movement Disorders Society. Both networks follow the same core protocol for patient recruitment and collection of data, imaging and biomaterials. Both networks host a web-based data registry and a central biorepository. Inclusion/exclusion criteria follow the 2017 Movement Disorder Society criteria for the clinical diagnosis of progressive supranuclear palsy.Results: Both networks started recruitment of patients by the end of 2015. As of November 2020, N = 354 and 269 patients were recruited into the DescribePSP and the ProPSP studies, respectively, and N = 131 and 87 patients received at least one follow-up visit.Conclusions: The DescribePSP and ProPSP networks are ideal resources for comprehensive natural history data of PSP, including imaging data and biological samples. In contrast to previous natural history studies, DescribePSP and ProPSP include not only patients with Richardson's syndrome, but also variant PSP phenotypes as well as patients at very early disease stages, before a diagnosis of possible or probable PSP can be made. This will allow for identification and evaluation of early biomarkers for diagnosis, prognosis, and progression.

Highlights

In 2015, two German multicenter research networks, DescribePSP and ProPSP, were set up by the authors with the ultimate goal to improve early clinical diagnosis, monitoring, and prediction of disease progression in patients with progressive supranuclear palsy (PSP).DescribePSP and ProPSP are acronyms
Clinico-pathological studies suggest that PSP has previously been underdiagnosed during lifetime and that the correct ante-mortem diagnosis of PSP has been delayed for several years, due to a lack of specific symptoms at early disease stages and due to heterogeneous clinical presentations [2, 4]
PSP, possible PSP, and probable PSP according to the MDSPSP criteria have been collected within both, the DescribePSP and the ProPSP networks

Summary

Introduction

In 2015, two German multicenter research networks, DescribePSP and ProPSP, were set up by the authors with the ultimate goal to improve early clinical diagnosis, monitoring, and prediction of disease progression in patients with progressive supranuclear palsy (PSP).DescribePSP and ProPSP are acronyms. Clinico-pathological studies suggest that PSP has previously been underdiagnosed during lifetime and that the correct ante-mortem diagnosis of PSP has been delayed for several years, due to a lack of specific symptoms at early disease stages and due to heterogeneous clinical presentations [2, 4]. Treatment strategies are presently restricted to symptomatic therapies, several tau targeting therapies are being developed for PSP [1] These developments further increase the need for correct and early clinical diagnosis of PSP and reliable prediction of disease progression, to set the stage for early disease-modifying interventions. The German research networks DescribePSP and ProPSP prospectively collect comprehensive clinical data, imaging data and biomaterials of patients with a clinical diagnosis of progressive supranuclear palsy. Clinical research into treatment strategies has been limited due to delay in clinical diagnosis and lack of natural history data on distinct clinical phenotypes

Methods

Results

Conclusion