Burden of Disease in Myasthenia Gravis

NCT ID: NCT03979521

Last Updated: 2022-01-04

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 1680 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2019-05-01
• Study Completion Date: 2019-07-31

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

Conditions

Myasthenia Gravis

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: RETROSPECTIVE

Eligibility Criteria

Inclusion Criteria

• Patients with Myasthenia Gravis
• Age ≥18 years

Exclusion Criteria

• none

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

NeuroCure Clinical Research Center, Charite, Berlin

OTHER

Sponsor Role: collaborator

Charite University, Berlin, Germany

OTHER

Sponsor Role: lead

Responsible Party

Andreas Meisel

Prof. Dr. med.

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Andreas Meisel, Prof. Dr. med.

Role: PRINCIPAL_INVESTIGATOR

Charite University, Berlin, Germany

Locations

NeuroCure Clinical Research Center (NCRC), Charité University, Berlin

Mitte, , Germany

Countries

Germany

References

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.

Reference Type: RESULT

PMID: 34800167 (View on PubMed)

Lehnerer S, Stegherr R, Grittner U, Stein M, Gerischer L, Stascheit F, Herdick M, Doksani P, Meisel A, Hoffmann S. The burden of disease in seronegative myasthenia gravis: a patient-centered perspective. Front Immunol. 2025 Apr 8;16:1555075. doi: 10.3389/fimmu.2025.1555075. eCollection 2025.

Reference Type: DERIVED

PMID: 40264778 (View on PubMed)

Lehnerer S, Herdick M, Mevius A, Grittner U, Gansel P, Joeres L, Biskup J, Meisel A. The economic burden of Myasthenia gravis from the patient s perspective and reflected in German claims data. Sci Rep. 2025 Feb 25;15(1):6687. doi: 10.1038/s41598-025-91372-7.

Reference Type: DERIVED

PMID: 39994434 (View on PubMed)

Stein M, Grittner U, Stegherr R, Gerischer L, Stascheit F, Hoffmann S, Herdick M, Legg D, Marbin D, Meisel A, Lehnerer S. The burden of myasthenia gravis - highlighting the impact on family planning and the role of social support. Front Neurol. 2023 Dec 14;14:1307627. doi: 10.3389/fneur.2023.1307627. eCollection 2023.

Reference Type: DERIVED

PMID: 38162439 (View on PubMed)

Other Identifiers

Mya-BoD

Identifier Type: -

Identifier Source: org_study_id