Fatigue, Myasthenia Gravis
Conditions
Keywords
Myasthenia Gravis, Fatigue, Digital features, Machine Learning Algorithms, Dysarthria, Dysphonia, Proximal arm fatigue, Ptosis
Brief summary
This study will make use of a cross-sectional design of MG patients and non-MG participants to quantitatively assess key MG symptoms, and to explore the applicability of machine learning algorithms to their measurement.
Detailed description
Due to the cross-sectional design, participants will only have to visit Leiden University Medical Center (LUMC) once. For patients already treated in the LUMC, we will try to align this visit with a standard clinical appointment. After inclusion, all baseline data, consisting of demographics, clinical history and a number of questionnaires (four for MG participants, three for non-MG participants), will be collected. The symptom-specific assessments are performed in a standard order, with the most fatiguing task (i.e. proximal arm fatigue static assessment) last. We estimate the visit will take a total of 60 minutes. This study is considered to be low risk. Withholding pyridostigmine for a limited period is part of standard care of MG (before investigations or clinical assessments) and does not affect long term clinical outcome. MG participants will consent to withhold pyridostigmine for 12 hours prior to the study visit if they are on this treatment and restart it after the visit. As this is a non-interventional, observational study where only questionnaire-based and non-contact digital data are being collected, the only source of marginal risk relates to data protection and confidentiality, including arrangements for the transfer and storage of data. Given it would not be possible to deidentify the digital audio or video data while maintaining the requisite integrity for data analysis, we will seek explicit consent for the sharing of this information in this identifiable format.
Interventions
None listed
Sponsors
Leiden University Medical Center
Huma
Study design
Observational model
CASE_CONTROL
Time perspective
CROSS_SECTIONAL
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
Yes
Inclusion criteria
1. Age ≥ 18 years 2. Ability to understand the requirements of the study and provide written informed consent. Inclusion Criteria for MG participants only: 1. A clinical diagnosis of myasthenia gravis (ocular or generalized) as defined by the Dutch national guideline (category "definite" or "probable" MG). 2. MGFA Clinical Classification of disease severity I-IV. 3. Subjects have at least one of the symptoms of interest (namely dysarthria, dysphonia, proximal arm fatigue and/or ptosis). Inclusion Criteria for non-MG participants only 1. Subjects are not diagnosed with and have no clinical suspicion of MG. 2. Subjects do not have a medical history of any of the symptoms of interest (namely dysarthria, dysphonia, proximal arm fatigue and/or ptosis).
Exclusion criteria
1. Not willing to be audio-recorded for the study assessments. 2. Not willing to be video-recorded for the study assessments. 3. Subjects currently taking part in a clinical trial of an Investigational Medicinal Product. 4. Subjects who have used an immediate release pyridostigmine-based medication in the 12 hours prior to their participation and participants on prolonged release pyridostigmine. 5. Subjects have cognitive or physical limitations that, in the opinion of the investigator, limits the subject's ability to complete study procedures/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Differentiating between MG-patients and non-MG participants using digital features of dysarhtria, dysphonia, proximal arm fatigue and ptosis. | Assessed at a single time point during outpatient visit | Using machine-learning algorithms. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Correlating digital features of dysarthria, dysphonia, proximal arm fatigue and ptosis in MG patients with disease severity as measured by the MGC score. | Assessed at a single time point during outpatient visit | Myasthenia Gravis Composite (MGC) is scored on an ordinal scale with four possible categories and weighted and consists of 10 items with a total score of 0-50; a higher score indicating more severe disease. |
| Correlating digital features of dysarthria, dysphonia, proximal arm fatigue and ptosis in MG patients with the impact of MG on daily activities as measured by the MG-ADL. | Assessed at a single time point during outpatient visit | MG Activities of Daily Living (MG-ADL) consists of 8 items with a total score of 0-24; a higher score indicating more severe disease. . |
| The performance of automated signal processing of speech recordings collected through smartphone microphone for detection of dysarthria and dysphonia compared to clinical assessment. | Assessed at a single time point during outpatient visit | Clinical assessment by two independent trained speech and language pathologists (SLP) using the Therapy Outcome Measure scale (TOMS) for dysarthria and GRBAS scale (Grade, Roughness, Breathiness, Asthenia and Strain) for dysphonia. |
| The performance of automated measurement of proximal arm-fatiguing exercises through computer vision techniques applied to smartphone camera recordings for detection of proximal arm muscle weakness and fatigability compared to clinical assessment. | Assessed at a single time point during outpatient visit | Clinical assessment by accredited clinicans. |
| The performance of automated measurement of ptosis-provoking exercises through computer vision techniques applied to smartphone camera recordings for detection of ptosis compared to clinical assessment. | Assessed at a single time point during outpatient visit | Clinical assessment by accredited clinicians. |
| Correlating digital features of dysarthria, dysphonia, proximal arm fatigue and ptosis in MG patients with their level of fatigue as measured by the CIS-fatigue subscale. | Assessed at a single time point during outpatient visit | Checklist Individual Strength (CIS-) fatigue subscale consists of 8 questions on fatigue experienced during the previous 2 weeks. Each question is scored on a 7-point Likert scale and a score ≥ 35 indicates severe fatigue. A higher score means more severe fatigue. The total score of the fatigue subscale ranges from 8-56 (the total score of the CIS questionnaire ranges from 20-140). |
Countries
Netherlands
Contacts
CONTACTMartijn R Tannemaat, MD, PhD
CONTACTYvonne JM Campman, MD
PRINCIPAL_INVESTIGATORMartijn R. Tannemaat, MD, PhD
Leiden University Medical Center
Outcome results
None listed