Effectiveness and Safety of Tacrolimus Combined With Low-dose Prednisone for Treatment of Myasthenia Gravis: A Real-world Study
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 160
- 试验地点
- 1
- 主要终点
- Change of MG-specific Activities of Daily Living scale (MG-ADL) from Baseline
研究概览
简要总结
This study is designed to evaluate the effectiveness and safety of tacrolimus combined with low-dose prednisone in the management of myasthenia gravis patients, compared to tacrolimus as initial immune monotherapy.
详细描述
This is a single center, observational real-world study recruiting myasthenia gravis patients from Neurology Departments of Xuanwu Hospital, aiming to compare effectiveness and safety of 2 different inmunotherapy for MG. The study plans to recruit 160 MG participants and divides into 2 treatment groups according to physician's judgment and preferences of patients, one is combined immunotherapy group in which tacrolimus added with low-dose prednisone (0.25mg/kg/d), and the other is tacrolimus monotherapy group. Both groups can be treated with pyridostigmine to relieve symptoms. Patients are followed up at 1, 3 and 6 month after treatment initiation to assess the efficacy of both regimen. The primary outcome is the change of MG-ADL scores. Also, liver and renal functions are tested to monitor any side effects. Patients' clinical records are uploaded to an online database.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical Diagnosis of MG is confirmed based on typical clinical features of fluctuating muscle weakness, with at least 1 of the following supporting evidence:
- •positive clinical response to acetylcholinesterase inhibitor
- •positive AchR-Ab or MuSK-Ab testing
- •decrement >10% in repetitive nerve stimulations study (RNS) or increased jitter on single-fibre electromyography (SFEMG)
- •MGFA clinical classification: I - IV
- •Baseline MG-ADL ≥ 3
- •Disease course from onset to enrollment ≤ 12 months
- •Cooperation to followup
- •Written informed consent
排除标准
- •Initiation of immunosuppressant for MG prior to screening, including Prednisone, Methylprednisolone, Azathioprine, Methotrexate, Cyclosporine A, Mycophenolate Mofetil, Tacrolimus and Cyclophosphamide
- •Treatment of immunosuppressant for other concomitant disease 6 months prior to recruitment
- •Rapid immunosuppressive treatments like Intravenous immunoglobulin or plasma exchange 1 month prior to recruitment
- •Thymectomy within 3 months prior to Screening
- •Concomitant chronic degenerative, psychiatric, or neurologic disorder that can cause weakness or fatigue
- •Consciousness, dementia or schizophrenia
- •Pregnancy or lactation, unwillingness to avoid pregnancy
- •Uncontrolled hypertension or diabetes, Liver or kidney dysfunction, Cataract, Severe osteoporosis, Femoral head necrosis; Hyperkalemia, HIV, Acute or chronic infection
- •Other conditions that would preclude participation
研究组 & 干预措施
Combined Immunotherapy
MG patients are treated with tacrolimus combined with low-dose prednisone (0.25mg/kg/d).
Symptomatic treatment like pyridostigmine bromide can be added to relieve symptoms (≤480mg/d).
干预措施: Pyridostigmine, Prednisone, Tacrolimus (Drug)
Tacrolimus monotherapy
MG patients are treated with tacrolimus as initial immune monotherapy. Symptomatic treatment like pyridostigmine bromide can be added to relieve symptoms (≤480mg/d).
干预措施: Pyridostigmine, Tacrolimus (Drug)
结局指标
主要结局
Change of MG-specific Activities of Daily Living scale (MG-ADL) from Baseline
时间窗: Baseline, 1 month, 3 months, 6 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.
次要结局
- Change of Myasthenia Gravis Quantity-of-Life Scale (MG-QoL15) from Baseline(Baseline, 1 month, 3 months, 6 months)
- Changes of MG-ADL subscores from baseline(Baseline, 1 month, 3 months, 6 months)
- Time to achievement of minimal manifestations (MMS) or better(From Baseline to 6 months)
- Change of Quantitative Myasthenia Gravis (QMG) Scores from Baseline(Baseline, 1 month, 3 months, 6 months)
- Withdrawal(Baseline to 6 months)
- Time to achievement of Patient-Acceptable Symptom States(From Baseline to 6 months)
- Serum IL-2 level(Baseline, 1 month, 3 months, 6 months)
- Treatment Failure(Baseline to 6 months)