Brief Title
Systematic Assessment of Laryngopharyngeal Function in Patients With MSA, PD, and 4repeat Tauopathies
Official Title
Prospective Observational Study for the Systematic Assessment of Laryngopharyngeal Function in Patients With Multiple System Atrophy, Parkinson's Disease and 4repeat Tauopathies
Brief Summary
This is a non-interventional observational study designed to systematically record the
results of routine laryngeal examinations and specific characteristics of dysphagia in
patients with multiple system atrophy (MSA), Parkinson's disease (PD) and progressive
supranuclear palsy (PSP) and related 4repeat tauopathies. The results of a fiberoptic /
flexible endoscopic evaluation of swallowing (FEES) while performing a structured task
protocol will be recorded. If available, laryngeal electromyography (EMG) results will also
be recorded. In addition to the examination results, demographic and disease-specific data
are collected, and two questionnaires, the Swallowing Disturbance Questionnaire for
Parkinson's Disease (SDQ-PD) and the swallowing specific Quality Of Life Questionnaire
(SWALQOL), are administered.
Detailed Description
Multiple system atrophy (MSA) is a sporadic progressive neurodegenerative disorder caused by
oligodendroglial aggregation of α-synuclein affecting predominantly the nigrostriatal,
olivo-ponto-cerebellar, and autonomic systems,resulting in a clinical presentation of
dysautonomia combined with either predominantly parkinsonian (MSA-P) or cerebellar (MSA-C)
symptoms of varying severity.In its early stage, the diagnosis of MSA according to the second
consensus criteria can be challenging. Therefore, the Movement Disorders Society MSA study
group recently addressed the importance of developing valuable diagnostic tools for securing
an early diagnosis in patients with MSA not only to estimate disease prognosis but also to
early initiate novel, potentially disease-modifying treatments in clinical trials. Despite
laryngopharyngeal dysfunction being associated with decreased life expectancy and quality of
life, systematic assessment of these functions in MSA is scarce. Previously, an
easy-to-implement MSA-FEES task-protocol was suggested to systematically assess
laryngopharyngeal function.
A pilot study on 8 patients with MSA not only showed that the task protocol was feasible and
well tolerated, but also that laryngopharyngeal symptoms where highly prevalent despite the
lack of clinical presentation (Warnecke et. al 2019). Moreover, irregular arytenoid
cartilages movements where present in all MSA-patients when performing this task protocol,
suggesting this symptom could serve as a clinical marker to identify MSA-patients.
Following this pilot study, an observational two center study assessed 57 MSA patients with
this protocol and compared findings to an age-matched cohort of PD-patients (Gandor et al.
2020). While only 43.9% of MSA patients had clinical symptoms of laryngeal dysfunction, 93%
showed laryngeal abnormalities during FEES performing the task-protocol. 91.2% of
MSA-patients showed irregular arytenoid cartilages movements. In contrast, only one PD
patient showed laryngeal abnormalities with vocal fold motion impairment, but not irregular
arytenoid cartilages movements. This study suggests that irregular arytenoid cartilages
movements allow differentiating MSA from PD with a sensitivity of 0.9 and a specificity of
1.0.
The aim of this FEEMSA trial is to continue recruitment of patients with MSA and PD and
systematically assess laryngopharyngeal function in an even larger cohort. Moreover, patients
with PSP and related 4repeat tauopathies will also be recruited at eligible sites to compare
results from this cohort to results in MSA and PD. If available, laryngeal EMG will also be
recorded.
Study Type
Observational
Primary Outcome
laryngeal movement disorders
Condition
Multiple System Atrophy
Study Arms / Comparison Groups
Multiple System Atrophy
Description: Patients diagnosed will probable or possible MSA according to the 2nd criteria for the diagnosis of MSA (Gilman 2008) that received laryngopharyngeal assessment according to the systematic task protocol during FEES (Warnecke et al. 2019).
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Estimated Enrollment
200
Start Date
September 1, 2017
Completion Date
July 31, 2023
Primary Completion Date
December 31, 2022
Eligibility Criteria
Inclusion Criteria:
- diagnosis of probable or possible multiple system atrophy according to current
consensus criteria (Gilman et al. 2008) or
- diagnosis of probable or possible PSP according to the the Movement Disorders Society
(MDS) diagnostic criteria (Höglinger et al. 2017) or
- diagnosis of Parkinson's disease according to the MDS diagnostic criteria (Postuma et
al 2015)
- Hoehn and Yahr Stage within the range of I-V
Exclusion Criteria:
- Patients who do not sign the consent form
- Patients who have contraindications for performing fiber endoscopic laryngoscopy and
swallowing examination.
- Pregnancy in female patients
Gender
All
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Florin Gandor, MD, +493320422781, [email protected]
Location Countries
Austria
Location Countries
Austria
Administrative Informations
NCT ID
NCT04706234
Organization ID
S21(a)/2017
Responsible Party
Principal Investigator
Study Sponsor
Kliniken Beelitz GmbH
Collaborators
University Hospital Muenster
Study Sponsor
Florin Gandor, MD, Principal Investigator, Movement Disorders Hospital Beelitz-Heilstätten, Germany
Verification Date
September 2021