Clinical trial NCT03979521: Burden of Disease in Myasthenia Gravis
Official title: Questionnaire Survey on the Systematic Recording and Characterization of Quality of Life Limitations in Myasthenia Gravis
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Myasthenia Gravis
Keywords: Myasthenia Gravis
Quality of life
Burden of disease
Social support
Lead sponsor: Charite University, Berlin, Germany
Sponsor class: OTHER
Collaborators: name: NeuroCure Clinical Research Center, Charite, Berlin
First posted: 2019-06-07
Enrollment: 1680
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients with Myasthenia Gravis being member at the German myasthenia gravis socitey (Deutsche Myasthenie Gesellschaft)

Brief summary: Questionnaire-based survey addressed to german patients with the chronic autoimmune muscle disease called myasthenia gravis regarding quality of life, socioeconomic impact, social support, course of disease, complications of therapy and psychological comorbidities.

Detailed description: Background:

Myasthenia gravis is a rare, chronic, antibody-mediated autoimmune disease that results in muscular weakness. The patients suffer from symptoms such as visual disturbances (especially double vision), weakness in the arms and legs, chewing and swallowing disorders and respiratory disorders, as well as crisis-related worsening of ventilation. This is often accompanied by pronounced fatigue (physical) and mental fatigue (depression).

Despite drug therapies that are on one hand symptomatic improving muscle strength, and on the other hand modulating the immune system, as well as specific procedures and drugs used in crises (plasmapheresis, immunoglobins) or refractory patients, patients are often severely limited, affecting everyday activities of self-care, family, social and professional life, thus affecting the mood and quality of life of patients. There is a lack of data measuring quality of life adequately.

Aim:

The aim of the data collection is to record the quality of life of myasthenia patients. By clinical information on the disease (including symptoms, course and therapy) and recording the social and occupational situation, the data should be contextualized. The following hypotheses are to be confirmed:

Primary hypotheses

* Myasthenia gravis affects the quality of life of patients comparable to neurological diseases such as multiple sclerosis
* Patients are limited in their participation in their private and professional life
* The severity of the disease is associated with the quality of life. Secondary hypotheses
* Myasthenia is associated with income busses and other economic disadvantages of the night
* Quality of life in myasthenia patients is related to fatigue and depression
* Quality of life in myasthenia patients depends on different therapy methods
* Myasthenia gravis has an impact on family planning and care for children.

Methods:

In cooperation with the German Myasthenia Gravis Society, a questionnaire prepared by the Charité will be sent to its members with a request for completion and an anonymous return. Similar projects have already been implemented in the past (response rate\> 30%). About 3,300 patients should be contacted.

The returned questionnaires are then statistically evaluated and presented as part of a publication. For the evaluation of the hypotheses suitable descriptive, univariate analysis methods are used. Furthermore, with sufficient case numbers, multivariate analyzes (e.g., logistic regression) are also planned.

The questionnaire contains general information and questions about:

* Education and employment
* Social situation
* Social Support
* Economic aspects
* Symptoms and severity, course, treatment of myasthenia
* Quality of life
* Fatigue and depression

Eligibility criteria: Inclusion Criteria:

* Patients with Myasthenia Gravis
* Age ≥18 years

Exclusion Criteria:

* none

Primary outcomes:
measure: Questionnaire for self-completion by patients containing questions about general information, information on myasthenia, information socioeconomic situation
timeFrame: directly after inclusion in the study
measure: Hospital Anxiety and Depression Scale (HADS)
timeFrame: directly after inclusion in the study
measure: Mya Quality of Life 15 (Mya QoL15)
timeFrame: directly after inclusion in the study
measure: Medical Outcomes Study Questionnaire Short Form 36 Health Survey (SF36)
timeFrame: directly after inclusion in the study
measure: Myasthenia gravis Activities of daily living (MG ADL)
timeFrame: directly after inclusion in the study
Secondary outcomes:
Other outcomes:
Locations:
city: Berlin-Mitte
country: Germany
facility: NeuroCure Clinical Research Center (NCRC), Charité University, Berlin
Study officials:
affiliation: Charite University, Berlin, Germany
name: Andreas Meisel, Prof. Dr. med.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Lehnerer S, Jacobi J, Schilling R, Grittner U, Marbin D, Gerischer L, Stascheit F, Krause M, Hoffmann S, Meisel A. Burden of disease in myasthenia gravis: taking the patient's perspective. J Neurol. 2022 Jun;269(6):3050-3063. doi: 10.1007/s00415-021-10891-1. Epub 2021 Nov 20.