Clinical Prognosis and Progression of Myasthenia Gravis Patients

Sponsor

Da, Yuwei, M.D. (Other)

Overall Status

Recruiting

CT.gov ID

NCT04101578

Collaborator

(none)

2,000

Enrollment

Location

94.7

Anticipated Duration (Months)

21.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Myasthenia Gravis	Drug: Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg)

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 Design

Study Type:

Observational

Anticipated Enrollment :

2000 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Prospective Observational Trial to Evaluate Clinical Prognosis and the Risk Factors for Progression for Myasthenia Gravis Patients

Actual Study Start Date :

Feb 8, 2017

Anticipated Primary Completion Date :

Jun 1, 2023

Anticipated Study Completion Date :

Dec 31, 2024

Arms and Interventions

Arm	Intervention/Treatment
Ocular MG Patients with autoimmune MG whose symptoms restricted to extraocular muscles	Drug: Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg) Treatment regimens are determined according to the physician's judgment and preferences of the patients. Other Names: Pyridostigmine Bromide, Prednisone, Methylprednisolone, Azathioprine, Tacrolimus, Cyclosporin A, Cyclophosphamide, Mycophenolate Mofetil, Methotrexate
Generalized MG Patients not only suffer from extraocular muscles weakness but also from limb weakness, bulbar symptoms, or even respiratory failure	Drug: Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg) Treatment regimens are determined according to the physician's judgment and preferences of the patients. Other Names: Pyridostigmine Bromide, Prednisone, Methylprednisolone, Azathioprine, Tacrolimus, Cyclosporin A, Cyclophosphamide, Mycophenolate Mofetil, Methotrexate

Arm

Intervention/Treatment

Ocular MG

Patients with autoimmune MG whose symptoms restricted to extraocular muscles

Drug: Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg)

Treatment regimens are determined according to the physician's judgment and preferences of the patients.

Other Names:

Pyridostigmine Bromide, Prednisone, Methylprednisolone, Azathioprine, Tacrolimus, Cyclosporin A, Cyclophosphamide, Mycophenolate Mofetil, Methotrexate

Generalized MG

Patients not only suffer from extraocular muscles weakness but also from limb weakness, bulbar symptoms, or even respiratory failure

Drug: Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg)

Treatment regimens are determined according to the physician's judgment and preferences of the patients.

Other Names:

Pyridostigmine Bromide, Prednisone, Methylprednisolone, Azathioprine, Tacrolimus, Cyclosporin A, Cyclophosphamide, Mycophenolate Mofetil, Methotrexate

Outcome Measures

Primary Outcome Measures

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

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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

15 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Age >14.
Clinical Diagnosis of MG with supporting evidence:
unequivocal clinical response to pyridostigmine
positive antibody testing
decrement >10% in repetitive nerve stimulations study (RNS) .
Willingness to sample collection, imaging study and other disease-related examinations and assessments.
Patients with informed consent.

Exclusion Criteria:

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