Prospective Observational Trial to Evaluate Clinical Prognosis and the Risk Factors for Progression for Myasthenia Gravis Patients
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 2,000
- 试验地点
- 1
- 主要终点
- Conversion rates from ocular to generalized MG at the last visit and risk factors.
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 15 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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 ≤
- •Severe systemic illness with life-expectancy less than 4 years.
- •Unwillingness to consent for collection of biological samples.
- •Inability to provide informed consent.
研究组 & 干预措施
Ocular MG
Patients with autoimmune MG whose symptoms restricted to extraocular muscles
干预措施: Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg) (Drug)
Generalized MG
Patients not only suffer from extraocular muscles weakness but also from limb weakness, bulbar symptoms, or even respiratory failure
干预措施: Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg) (Drug)
结局指标
主要结局
Conversion rates from ocular to generalized MG at the last visit and risk factors.
时间窗: 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.
时间窗: 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).
时间窗: 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.
时间窗: 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).
次要结局
- Proportion of Patients with Treatment-related Adverse Experiences.(3 months, 6 months, 12 months, 24 months, 36 months, 48 months)
- Changes in titers of MG antibodies.(Baseline, 12 months, 24 months, 36 months, 48 months)