Clinical prognostic factors in progressive supranuclear palsy: Implications for clinical trials
- PMID: 39957197
- DOI: 10.1177/1877718X241291996
Clinical prognostic factors in progressive supranuclear palsy: Implications for clinical trials
Abstract
Background: Progressive supranuclear palsy (PSP) is a rare neurodegenerative disease with diverse clinical phenotypes, prompting the development of new diagnostic criteria known as the MDS-PSP classification. However, little is known about the prognostic value of this classification in order to better stratify patients for the clinical trials.
Objective: To assess the impact of the different clinical phenotypes according to the MDS-PSP classification on prognosis using the clinical milestones of death, severe dysphagia, institutionalization, and need for walking aid.
Methods: A prospective cohort of 205 PSP patients from Lille University Hospital was analyzed retrospectively. Patients were classified into different MSD-PSP phenotypes according to their clinical presentation after 3 years of follow-up. The milestones of death, severe dysphagia, institutionalization, and need for walking aid were recorded, and a survival analysis was performed to describe the prognosis of each disease presentation.
Results: Median survival time was 6.4 (interquartile range (IQR): 4.8-8.6) years and mean diagnostic delay from symptom onset was 38.1 ± 22.5 months. PSP Richardson Syndrome (PSP-RS) had a poorer survival rate and a higher occurrence of severe dysphagia and need for walking aid compared to PSP variants such as PSP Parkinsonism (PSP-P), PSP postural instability without ocular motor dysfunction (PSP-PI), and other rare phenotypes.
Conclusions: PSP-RS has a less favorable prognosis compared to PSP variants stratified according to the MDS-PSP classification. This classification could assist in selecting patients for clinical trials and help design outcomes that account for the disease heterogeneity.
Keywords: MDS-PSP classification; atypical parkinsonism; prognosis; progressive supranuclear palsy; survival analysis.
Plain language summary
Progressive supranuclear palsy (PSP) is a rare and debilitating neurodegenerative disease marked by diverse combination of motor and cognitive symptoms. No treatment is currently available to slow down disease progression. The multiple clinical presentations of PSP compromise clinical trials. Thus, we aimed to study the differential prognosis of the different forms of the disease. We showed that PSP-Richardson Syndrome (PSP-RS), which is the most frequent presentation, seems to have a poorer prognosis than other variants on survival and other disability criteria such as severe dysphagia occurrence. This study suggests that these differences should account to improve the design of future clinical trials.
Conflict of interest statement
Declaration of conflicting interestsThe author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Luc Defebvre served on the Scientific Advisory Board for Abbvie and has received honoraria from pharmaceutical companies for consultancy and lectures including Abbvie, Novartis, Aguettant, and Orkyn.Stéphanie Bombois received honoraria from Biogen for a symposium (2021) and from Esai for a lecture (2020), and is an investigator without personal fees in therapeutical trials for Alzheimer's disease from Biogen, Roche, Eisai, Eli Lilly, Janssen, Johnson & Johnson, Alector, NovoNordisk, Genentech, AB science, and Novartis.David Devos has led four investigator-driven studies funded by academic grants from France from the French Ministry of Health and Research and the European Commission Horizon 2020 PHC13 2014–2015 (N° 633190), involving deferiprone, provided free of charge by ApoPharma Inc. and Chiesi Canada Corp. (FAIRPARK-I, FAIRPARK-II and SAFE-FAIR ALS-I, FAIR-ALS-II). He has served on advisory boards, served as a consultant and given lectures for pharmaceutical companies such as Orkyn, Abbvie, Medtronic, and Boston Scientific.Caroline Moreau has received grants from the France Parkinson & Vaincre Parkinson charities, and French ANR. She has received various honoraria from pharmaceutical companies for consultancy and lectures on Parkinson's disease at symposia, including Orkyn, Elivie, Abbvie, and Boston Scientific. She is an Editorial Board member of this journal, but was not involved in the peer-review process of this article nor had access to any information regarding its peer review.All other authors declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article.
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