Mycophenolate Mofetil Treatment With Neuromyelitis Optica Spectrum Disorders in Chinese Patients

Study Overview
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Brief Summary
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Neuromyelitis optica (NMO) is an autoimmune inflammatory demyelinating disease of the central nervous system that leads to blindness and paralysis. Since disability accrues incrementally related to attacks, attack prevention with immunosuppressive therapy is the mainstay of preventing disability. However, there is no standard immunosuppressive treatment strategy for NMO relapse prevention. In a previous study, the investigators provided evidence supporting the use of azathioprine plus a low dose corticosteroid as an effective strategy which is associated with a reduction in the risk of relapse in Chinese patients with NMO, but azathioprine has bone marrow suppression and other side effects. Mycophenolate mofetil (MMF) is a new immunosuppressant with rapid onset, fewer side effects and other advantages. In recent years, MMF has been used in different immune-related neurological diseases; some literature shown the possible efficacy of MMF in NMO treatment. In this research, a multi-center (Third Affiliated Hospital of Sun Yat-sen University, Zhongshan Ophthalmic Centre of Sun Yat-sen University, Nangfang Hospital of Southern Medical University) study will carry out to evaluate the efficacy and safety of mycophenolate mofetil therapy in NMO spectrum disorders.

Official Title
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Mycophenolate Mofetil Treatment With Neuromyelitis Optica Spectrum Disorders in Chinese Patients

Conditions
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Neuromyelitis Optica Spectrum Disorders, Mycophenolate Mofetil, Efficacy and Safety

Intervention / Treatment
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* Drug: Mycophenolate mofetil
* Drug: Prednisone


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Meet the 2006 Wingerchuk diagnostic criteria; NMO-immunoglobulin G seropositive; Between 18 to 65 years old; Relapse: more than 2 relapses in recent 2 years; more than 1 relapses in recent 1 years; Expanded disability status scale: expanded disability status scale≤7.0, and visual acuity ≥20 / 100 at least in one eye Understand the purpose and procedures of this study, and written informed consent is obtained. Exclusion Criteria: Using immunosuppressive agents and other drugs affect evaluation, and drug withdrawal less than 3 months; Patients with any of the following diseases: transaminases elevation exceed 2 times of the normal upper limit; white blood cell <4 × 109 / L, Hemoglobin <80g / L, platelet <100 × 109 / L; With serious cardiovascular, liver, kidneys and other vital organs and blood, endocrine system diseases, cancer history; With immunodeficiency, uncontrolled infection and active gastrointestinal diseases (such as gastric ulcer, etc.); Pregnancy, breast-feeding women and male or female who plans to conceive recently; Allergy to mycophenolate mofetil and prednisone.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Mycophenolate mofetil plus prednisone<br>Mycophenolate mofetil 500mg Bid and prednisone 10mg Qd | Drug: Mycophenolate mofetil<br>* Mycophenolate mofetil 500mg Bid<br>Drug: Prednisone<br>* prednisone 10mg Qd<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Annualized relapse rate day 360 | Patients come back for follow-up visit on day 360 after staring treatment, and annualized relapse rate was evaluated. | day 360 after staring treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| expanded disability status scale | Patients come back for follow-up visit on day 1, 14, 30, 90, 180, 270, 360 after staring treatment, and expanded disability status scale was evaluated. | day 1, 14, 30, 90, 180, 270, 360 after staring treatment | 
| Hauser scale | Patients come back for follow-up visit on day 1, 14, 30, 90, 180, 270, 360 after staring treatment, and Hauser scale was evaluated. | day 1, 14, 30, 90, 180, 270, 360 after staring treatment | 
| vision scale | Patients come back for follow-up visit on day 1, 14, 30, 90, 180, 270, 360 after staring treatment, and vision scale was evaluated. | day 1, 14, 30, 90, 180, 270, 360 after staring treatment | 
| Lesions in brain and spinal cord | Patients come back for follow-up visit on day 1, 14, 30, 90, 180, 270, 360 after staring treatment, lesions in brain and spinal cord were evaluated by MRI. | day 1, 14, 30, 90, 180, 270, 360 after staring treatment | 
| Annualized relapse rate | Patients come back for follow-up visit on day 1, 14, 30, 90, 180, 270 after staring treatment, and annualized relapse rate was evaluated. | day 1, 14, 30, 90, 180, 270 after staring treatment |