Screening in Oculopharyngeal Muscular Dystrophy

Study Overview
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Brief Summary
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Oculopharyngeal muscular dystrophy (OPMD) is a rare myopathic disease that results in progressive degeneration of the oral and pharyngeal muscular, resulting in severe dysphagia and dysarthria. OPMD is considered a rare disease; therefore, limited research is available on the natural progression of the disease or the utility of biomarkers to identify swallowing impairment. The aim of this study is: To identify accurate, reliable and non-invasive clinical markers of swallowing impairment To determine the discriminate ability of these markers to identify impairments in swallow safety and swallowing efficiency.

Detailed Description
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Participants will be recruited from the Neurology clinic at the University of Florida. The single evaluation will occur in the PIs research laboratory at the University of Florida, Gainesville, Florida.

Official Title
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Determination of Accurate Screening Tools for Dysphagia in Oculopharyngeal Muscular Dystrophy

Conditions
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Oculopharyngeal Muscular Dystrophy

Intervention / Treatment
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* Procedure: Videofluoroscopic swallowing study
* Other: Functional Oral Intake Scale
* Other: Eating Assessment Tool-10


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: diagnosis of oculopharyngeal muscular dystrophy no allergies to barium or capsaicin, no tracheotomy or mechanical ventilation no significant concurrent respiratory disease (e.g., COPD). Exclusion Criteria: Pregnant Women

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Screening<br>Participants enrolled will undergo testing of the swallowing mechanism | Procedure: Videofluoroscopic swallowing study<br>* Videofluoroscopic swallowing study will be performed to measure the oropharyngeal swallowing.<br>* Other names: modified barium swallow (MBS);Other: Functional Oral Intake Scale<br>* The Functional Oral Intake Scale (FOIS) will be used for participants to report their food intake habits.<br>* Other names: FOIS;Other: Eating Assessment Tool-10<br>* The Eating Assessment Tool-10 will be used for participant reporting of swallowing system severity.<br>* Other names: EAT-10;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Dynamic Imaging Grade of Swallowing Toxicity | Global Metric of swallowing Safety and Efficiency | Baseline | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Dietary intake as confirmed by Functional Oral Intake Scale (FOIS) | FOIS is a validated 7-point scale indexing an individual's ability to intake food on a day-to-day basis. TUBE DEPENDENT (levels 1-3) No oral intake Tube dependent with minimal/inconsistent oral intake Tube supplements with consistent oral intake TOTAL ORAL INTAKE (levels 4-7) Total oral intake of a single consistency Total oral intake of multiple consistencies requiring special preparation Total oral intake with no special preparation, but must avoid specific foods or liquid items Total oral intake with no restrictions | Baseline | 
| Participant perception of swallowing related symptoms as confirmed by The Eating Assessment Tool 10 (EAT-10) | EAT-10 is a 10-item validated self-administered dysphagia severity symptom survey. Score is 0 - 40 with 0 being the best possible score and 40 the worst possible score. | Baseline | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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screening, OPMD, dysphagia, dysarthria