Benatar M, Kaminski H
Emory University, Neurology Department, 1365A Clifton Road NE, Atlanta, GA 30322, USA.
Cochrane Database Syst Rev. 2006 Apr 19(2):CD005081. doi: 10.1002/14651858.CD005081.pub2.
Approximately 50% of people with myasthenia gravis present initially with purely ocular symptoms, so called ocular myasthenia and between 50 to 60% of these people will progress to develop generalized disease. The vast majority will do so within the first one to two years. There is controversy surrounding the appropriate management of patients with ocular myasthenia.
To perform a systematic review of the literature relevant to the treatment of ocular myasthenia and to answer three specific questions. Are there any medical or surgical treatments that have an impact on the risk of progression from ocular to generalized myasthenia gravis? Are there any medical or surgical treatments that improve symptoms of diplopia or ptosis in ocular myasthenia? What is the frequency of side effects associated with treatments used in people with ocular myasthenia?
We searched the Cochrane Neuromuscular Disease Group Trials Register (searched December 2004), MEDLINE (1996 to 2004) and EMBASE (1980 to 2004) for randomized controlled trials as well as case-control and cohort studies. The titles and abstracts of all articles were read by both authors and the full text of all articles that were of possible relevance was reviewed in full. The references of all manuscripts included in the review were scanned to identify additional articles of relevance and experts in the field were contacted to identify additional published and unpublished data. Where necessary and possible, we contacted authors for further information.
To be included in the review, studies had to meet three criteria: (a) randomized (or quasi-randomized) controlled study design; (b) active treatment compared to placebo, no treatment or some other treatment; and (c) results reported separately for patients with ocular myasthenia (grade 1) as defined by the Myasthenia Gravis Foundation of America.
We collected data regarding the risk of progression to generalized myasthenia gravis, improvement in ocular symptoms, and the frequency of treatment-related side effects.
We identified two randomized controlled trials relevant to the treatment of ocular myasthenia, only one of which reported results in terms of the pre-specified outcome measures used in this review. This study included only three participants and was of limited methodological quality. In the absence of data from randomized controlled trials, we present a review of the available observational data.
AUTHORS' CONCLUSIONS: There are no data from randomized controlled trials on the impact of any form of treatment on the risk of progression from ocular to generalized myasthenia gravis. The available randomized controlled literature does not permit any meaningful conclusions about the efficacy of any form of treatment for ocular myasthenia. Data from several reasonably good quality observational studies suggest that corticosteroids and azathioprine may be beneficial in reducing the risk of progression to generalized myasthenia gravis.
约50%的重症肌无力患者最初仅表现为眼部症状，即所谓的眼肌型重症肌无力，其中50%至60%的患者会进展为全身型疾病。绝大多数患者会在最初的一到两年内出现这种情况。眼肌型重症肌无力患者的恰当治疗方法存在争议。
对与眼肌型重症肌无力治疗相关的文献进行系统综述，并回答三个具体问题。是否存在对眼肌型重症肌无力进展为全身型重症肌无力风险有影响的内科或外科治疗方法？是否存在改善眼肌型重症肌无力复视或上睑下垂症状的内科或外科治疗方法？眼肌型重症肌无力患者所使用治疗方法相关的副作用发生频率是多少？
我们检索了Cochrane神经肌肉疾病组试验注册库（2004年12月检索）、MEDLINE（1996年至2004年）和EMBASE（1980年至2004年），以查找随机对照试验以及病例对照和队列研究。两位作者阅读了所有文章的标题和摘要，并对所有可能相关文章的全文进行了全面审查。对综述中纳入的所有手稿的参考文献进行了筛选，以识别其他相关文章，并联系该领域的专家以识别其他已发表和未发表的数据。必要且可能时，我们联系作者以获取更多信息。
要纳入综述，研究必须符合三个标准：（a）随机（或半随机）对照研究设计；（b）与安慰剂、不治疗或其他某种治疗相比的积极治疗；（c）按美国重症肌无力基金会定义，分别报告眼肌型重症肌无力患者（1级）的结果。
我们收集了关于进展为全身型重症肌无力风险、眼部症状改善情况以及治疗相关副作用发生频率的数据。
我们识别出两项与眼肌型重症肌无力治疗相关的随机对照试验，其中只有一项按照本综述中预先设定的结局指标报告了结果。该研究仅纳入了三名参与者，且方法学质量有限。由于缺乏随机对照试验的数据，我们对现有的观察性数据进行了综述。
尚无随机对照试验数据表明任何形式的治疗对眼肌型重症肌无力进展为全身型重症肌无力的风险有影响。现有的随机对照文献无法就任何形式的眼肌型重症肌无力治疗的疗效得出有意义的结论。来自几项质量相当不错的观察性研究的数据表明，皮质类固醇和硫唑嘌呤可能有助于降低进展为全身型重症肌无力的风险。
