Clinical Prognosis and Progression of Myasthenia Gravis Patients

Recruiting

• Conditions: Myasthenia Gravis
• Interventions: Drug: Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg)

• Registration Number: NCT04101578
• Lead Sponsor: Da, Yuwei, M.D.

Brief Summary

This study collects the clinical data of myasthenia gravis (MG) patients, assesses outcomes and adverse effects of different treatment regimens, and searches for risk factors of conversion to generalized MG.

Detailed Description

This is a multicenter, observational cohort trial in the real-world clinical setting recruiting MG patients from Neurology Departments of 6 hospitals in different regions of China. Clinical manifestations, laboratory test results, chest imaging and history of thymectomy are recorded. Patients will be classified by clinical manifestation as well as antibody status, and treatment regimens are determined according to the physician's judgment and preferences of the patients. Patients are followed up prospectively on regular to assess the outcomes of treatments and monitor any side effects. Peripheral blood samples are collected annually. Patients' clinical records are uploaded to an online database. The investigators plan to recruit a final sample of 2000 patients for analysis.

Study Timeline

• Study Start: 2017-02-08
• Study First Submitted: 2019-09-16
• Study First Submitted QC: 2019-09-23
• Study First Posted: 2019-09-24
• Status Verified: 2021-08
• Last Update Submitted: 2021-08-19
• Last Update Posted: 2021-08-23
• Primary Completion: 2023-06-01
• Study Completion: 2024-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 2000

Inclusion Criteria

1. Age >14.

2. Clinical Diagnosis of MG with supporting evidence:

   1. unequivocal clinical response to pyridostigmine
   2. positive antibody testing
   3. decrement >10% in repetitive nerve stimulations study (RNS) .

3. Willingness to sample collection, imaging study and other disease-related examinations and assessments.

4. Patients with informed consent.

Exclusion Criteria

1. History of chronic degenerative, psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue.
2. Age ≤14 years.
3. Severe anxiety, depression or schizophrenia.
4. Cognitive impairment or mini-mental state examination (MMSE) score ≤24.
5. Severe systemic illness with life-expectancy less than 4 years.
6. Unwillingness to consent for collection of biological samples.
7. Inability to provide informed consent.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Ocular MG	Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg)	Patients with autoimmune MG whose symptoms restricted to extraocular muscles
Generalized MG	Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg)	Patients not only suffer from extraocular muscles weakness but also from limb weakness, bulbar symptoms, or even respiratory failure

Primary Outcome Measures

Name	Time	Method
Conversion rates from ocular to generalized MG at the last visit and risk factors.	Baseline, 48 months	Ocular MG patients are followed up to determine the ratio of conversion to generalized disease at the end of follow-up. The clinical records will be retrospectively analyzed to search for risk factors of progressing.
Change in Quantitative Myasthenia Gravis (QMG) Scores from Baseline to 48 months.	Baseline, 12 months, 24 months, 36 months, 48 months	The QMG is a 13-item scale which measures ocular, bulbar, limb function and respiratory function. The total score ranges from 0 (no myasthenic findings) to 39 (maximal myasthenic deficits) obtained by summing the responses to each individual item (None=0, Mild=1, Moderate=2, Severe=3).
Change in MG-specific Activities of Daily Living scale (MG-ADL).	Baseline,3months, 6 months, 9 months, 12 months, 18 months, 24 months, 30months, 36 months, 42 months, 48 months	The MG-ADL is an 8-item scale to assess symptoms of myasthenia gravis patients obtained by summing the responses to each individual item (Grades: 0,1,2,3). The score ranges from 0 to 24.
The proportion of patients reaching minimal manifestations (MM) or better.	48 months	Clinical statuses of patients are assessed and categorized according to Myasthenia Gravis Foundation of America (MGFA) postintervention status (PIS). MM or better includes Minimal Manifestation (MM), Pharmacologic Remission (PR) or Complete Remission (CR).

Secondary Outcome Measures

Name	Time	Method
Proportion of Patients with Treatment-related Adverse Experiences.	3 months, 6 months, 12 months, 24 months, 36 months, 48 months	Treatment-Related Adverse Events (AEs) are evaluated in patients of different regimens.
Changes in titers of MG antibodies.	Baseline, 12 months, 24 months, 36 months, 48 months	MG antibodies are detected at enrollment and the titers of antibodies will be monitored annually.

Trial Locations

Locations (1)

Xuan Wu Hospital, Capital Medical University; 🇨🇳 Beijing, Beijing, China