Evaluation of the Efficacy of a Two-week EMST on Dysphagia in Parkinsonian Patients

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Brief Title

Evaluation of the Efficacy of a Two-week EMST on Dysphagia in Parkinsonian Patients

Official Title

Evaluation of the Efficacy of a Two-week Expiratory Muscle Training on Swallowing Function in Patients With Parkinsonian Disorders

Brief Summary

      This is an interventional therapy study designed to evaluate the efficacy of a two-week
      intervention, i.e. training with a specialized exhalation training device (called expiratory
      muscle strength training; EMST150 or EMST75; Aspire Products, Gainsville, FL) on swallowing
      function in patients with neurodegenerative Parkinsonian disorders. This study involves a
      routine endoscopic evaluation of swallowing (FEES) to diagnose dysphagia before and after the
      intervention. Between the two FEES, a two-week exhalation training program takes place, which
      the patients perform independently following instructions from a speech and lanuage
      pathologist. In addition demographic and disease-specific data and two questionnaires
      (Swallowing Disturbance Questionnaire for Parkinson's disease patients, SDQ-PD, and
      Swallowing specific Quality Of Life Questionnaire SWAL-QoL) are recorded.
    

Detailed Description

      This is an interventional therapy study designed to evaluate the efficacy of a two-week
      intervention, i.e. training with a specialized exhalation training device (called expiratory
      muscle strength training; EMST150 or EMST75; Aspire Products, Gainsville, FL) on swallowing
      function in patients with neurodegenerative Parkinsonian disorders. This study involves a
      routine endoscopic evaluation of swallowing (FEES) to diagnose dysphagia before and after the
      intervention. Between the two FEES, a two-week exhalation training program takes place, which
      the patients perform independently following instructions from a speech and lanuage
      pathologist. In addition demographic and disease-specific data and two questionnaires
      (Swallowing Disturbance Questionnaire for Parkinson's disease patients, SDQ-PD, and
      Swallowing specific Quality Of Life Questionnaire SWAL-QoL) are recorded.

      Main hypothesis: Two weeks of EMST will lead to a significant improvement of the endoscopic
      dysphagia score in patients with neurodegenerative Parkinsonian disorders with endoscopically
      proven oropharyngeal dysphagia. Patients with idiopathic Parkinson's syndrome as well as its
      differential diagnoses, multiple system atrophy (MSA) and diseases from the group of 4repeat
      tauopathies, will be examined.

      Idiopathic Parkinson's disease (PD):

      More than 80% of all patients with PD develop clinically relevant dysphagia during the course
      of the disease. This can lead to a significant reduction in quality of life, decreased
      medication efficacy, malnutrition, dehydration, and ultimately aspiration pneumonia in
      affected patients, which is the most common cause of death in advanced PD patients. The cause
      of PD-associated dysphagia is a multifactorial genesis with impairment of dopaminergic and
      non-dopaminergic pathways of the central swallowing network and additional peripheral
      neuromuscular influences.

      Multiple System Atrophy (MSA):

      MSA is clinically associated with autonomic dysregulation manifested by neurogenic urinary
      bladder emptying dysfunction, orthostatic hypotension, sleep-related respiratory
      dysregulation, etc. in addition to Parkinsonian and/or cerebellar symptoms. Median survival
      after diagnosis is approximately 7 years. At disease onset, differentiation from idiopathic
      PD is difficult. However, relatively early in the course of the disease, symptoms such as a
      brittle voice or stridor may appear during normal breathing, which may be clues to the
      diagnosis of MSA. Since laryngeal abnormalities are associated with a significantly reduced
      life expectancy, they require special attention and diagnosis. In this regard, we have
      recently shown that a high prevalence of laryngeal movement abnormalities is present in MSA
      patients and can even be used to differentiate them from idiopathic Parkinson's disease.
      Swallowing function, the regulation of which is associated with autonomic centers of
      laryngeal function, is also often impaired early in patients with MSA. Because dysphagia can
      further limit quality of life and life expectancy, this symptom also requires early diagnosis
      and treatment. We could recently show that the dysphagia pattern also differs between PD and
      MSA.

      4repeat tauopathies (4RT): The form of 4RT, also named progressive supranuclear gaze palsy
      (PSP) with all clinical subtypes, is a rapidly progressive neurodegenerative disease that
      leads to progressive functional impairment of cortical and subcortical function in affected
      individuals due to accumulation of tau protein in the brain. Due to the clinical variability
      of presentation, early diagnostic certainty is desirable, especially since human IgG4-
      antibodies directed against extracellular tau protein are currently in clinical trials to
      modify the course of the disease (see NCT03068468, NCT02985879). 4RT are also associated with
      swallowing and speech problems, and aspiration pneumonia is among the leading causes of death
      in this disease group. In addition, characteristic abnormalities may also occur due to
      dystonic dysinnervation of laryngeal muscles.

      For dysphagia in atypical Parkinsonian syndromes, there are no proven interventional or drug
      therapy options. Previous studies in patients with PD had shown efficacy of a four-week
      training with a special expiratory muscle strength training (EMST) device. Both swallowing
      reliability and swallowing efficiency were improved. Studies on the efficacy of EMST on
      dysphagia of atypical Parkinsonian syndromes do not yet exist. Having demonstrated the
      efficacy of EMST in a 4-week intervention regime, the first aim is to test whether a
      shortened intervention duration of two weeks is also effective in patient with PD. In
      addition, this protocol will be performed in patients with MSA and 4RT to investigate
      effects, particularly on swallowing safety (reduction of penetration/aspiration events) and
      swallowing efficiency (reduction of pharyngeal residuals). Currently, all patients with PD,
      MSA, or 4RT at participating study sites receive a speech and language pathology examination
      and endoscopic diagnostics according to a defined protocol. In addition to phoniatric and
      swallowing-specific examinations, an endoscopic evaluation of swallowing (FEES) is routinely
      performed. Regarding the efficacy of EMST training on pharyngeal dysphagia in patients with
      PD, two randomized, placebo-controlled studies from recent years with n=60 and n=50 patients,
      respectively, have already shown an efficacy of training on both swallowing reliability and
      swallowing efficiency in PD patients. Relevant side effects or risks from training for the
      patients were not reported in the studies.

      Studies using an EMST device have been conducted in recent years not only in patients with
      IPS, but also in patients with dysphagia due to other primary pathologies (e.g., amyotrophic
      lateral sclerosis, multiple sclerosis, stroke, etc.). In summary, a positive effect was
      shown, particularly on laryngeal muscle strengthening, which was reflected in an improvement
      in dysphagia.

      The affected patients receive an initial endoscopic evaluation of the swallowing act in the
      clinical setting as part of routine diagnostics. In addition, oral and written patient
      education is provided. After written consent on the informed consent form, the following data
      are then collected anonymously prior to the start of the intervention:

        1. sociographic data

        2. disease milestones

        3. MDS-UPDRS I-IV

        4. UMSARS / PSP-RS

        5. SDQ-PD

        6. SWAL-QOL

        7. Hoehn and Yahr stage

        8. Current drug therapy

        9. MSA-FEES examination protocol

       10. Pulmonary function test

      This is followed by the individual adjustment of the EMST device for the respective patient.
      For this purpose, the maximum expiratory pressure (MEP) is first determined for the
      respective patient in the pulmonary function examination using a pressure manometer. 75% of
      the MEP is set for the subsequent training on the EMST device. This is then followed by the
      actual intervention. For this, the patient receives logopedic instruction on the correct use
      of the EMST device. The intervention regime then consists of 5x5 breaths per day for 14
      consecutive days. Immediately following, items 3-10 will be collected again.

        1. The collection of data regarding the effectiveness of EMST in a shortened protocol (two
           weeks) on swallowing function in patients with PD can help to integrate this form of
           therapy firmly in the therapy regime of speech and language therapy also in the context
           of inpatient stays.

        2. The evaluation of the efficacy of EMST in atypical Parkinsonian syndromes is the first
           interventional study with the attempt to improve dysphagia in these disease spectra. If
           this study demonstrates improvement in dysphagia in MSA and 4RT, appropriate patients
           would be offered a specific EMST in the future, which may then delay or prevent the
           development of higher-grade dysphagia. Since consecutive aspiration pneumonia on the
           floor of neurogenic dysphagia is the leading cause of death in these patients, this
           could at least delay serious medical complications in the future, improve quality of
           life, and prolong survival. In addition, new evidence would be gained for the use of
           activating therapies in the treatment of Parkinson's-associated dysphagia.
    


Study Type

Interventional


Primary Outcome

dysphagia-score

Secondary Outcome

 SDQ-PD score

Condition

Parkinson Disease

Intervention

expiratory muscle strentgh training (EMST)

Study Arms / Comparison Groups

 PD patients
Description:  patients diagnosed with PD will be allocated to this arm

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Device

Estimated Enrollment

75

Start Date

November 15, 2021

Completion Date

December 31, 2022

Primary Completion Date

August 31, 2022

Eligibility Criteria

        Inclusion Criteria:

          -  ≥ 18 years of age with.

          -  diagnosis of idiopathic Parkinson's disease according to the updated diagnostic
             criteria (Postuma 2015) or (3) Diagnosis of a possible or probable Multiple System
             Atrophy according to the diagnostic criteria (Gilman 2008) or (4) Diagnosis of a
             possible or probable progressive supranuclear gaze palsy according to the diagnostic
             criteria (Höglinger 2017) (5) in Hoehn and Yahr stages I-V.

        Exclusion Criteria:

          1. Patients that do not sign the informed consent form

          2. Patients who have contraindications for a fiberendoscopic swallowing examination

          3. Patients who have contraindications to the two weeks of EMST training (e.g., severe
             pulmonary disease, severe dementia).

          4. Patients who have competing causes of dysphagia (e.g., history of stroke, tumor in the
             neck).
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Florin Gandor, MD, +493320422781, [email protected]

Location Countries

Germany

Location Countries

Germany

Administrative Informations


NCT ID

NCT05139342

Organization ID

2021-2155-BO-ff


Responsible Party

Sponsor

Study Sponsor

Kliniken Beelitz GmbH


Study Sponsor

Florin Gandor, MD, Principal Investigator, Movement Disorders Hospital Beelitz-Heilstätten,


Verification Date

March 2022