Tacrolimus Combined With Low-dose Prednisone for Treatment of Myasthenia Gravis

Da, Yuwei, M.D.

Status: Enrolling

Conditions: Myasthenia Gravis

Treatments: Drug: Pyridostigmine, Prednisone, Tacrolimus; Drug: Pyridostigmine, Tacrolimus

Study type: Observational

Identifiers

NCT04768465

1.0

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 160 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Age ≥18

• Clinical Diagnosis of MG is confirmed based on typical clinical features of fluctuating muscle weakness, with at least 1 of the following supporting evidence:

  1. positive clinical response to acetylcholinesterase inhibitor
  2. positive AchR-Ab or MuSK-Ab testing
  3. 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

Exclusion criteria

• 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

Trial design

160 participants in 2 patient groups

Combined Immunotherapy

Description:

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).

Treatment:

Drug: Pyridostigmine, Prednisone, Tacrolimus

Tacrolimus monotherapy

Description:

MG patients are treated with tacrolimus as initial immune monotherapy. Symptomatic treatment like pyridostigmine bromide can be added to relieve symptoms (≤480mg/d).

Treatment:

Drug: Pyridostigmine, Tacrolimus