摘要
目的评价中剂量环磷酰胺（CTX）联合甲泼尼龙治疗重症肌无力（MG）危象的有效性与安全性。方法采用前瞻、开放、平行和随机对照的研究方法，选择1999年1月至2010年10月在中山大学附属第一医院神经科就诊的156例重症肌无力危象患者作为研究对象，其中CTX组与对照组各78例。CTX组予甲泼尼龙500mg／d连用3d，后改为250mg／d，每隔3d剂量减半，直到62．5mg／d改泼尼松30mg／d口服维持至试验结束。同时加用CTX0．4g静脉推注，连用3d后改为0．4g，隔3d一次。对照组单独应用甲泼尼龙。根据MG的临床绝对和相对评分法评定疗效。结果（1）CTX组中54例（69．2％）患者3d内呼吸困难消失，而对照组中36例（46．2％）患者在8～14d内呼吸困难消失，两组比较差异有统计学意义（P=0．000）。（2）CTX组中超过一半的患者在10～14d内肢体无力（44例，56％）与吞咽功能（47例，60．3％）得到明显的改善，而同样情况的对照组患者28d后症状改善。两组比较差异有统计学意义（P=0．000）。（3）CTX组中，54例（69．2％）MG患者应用CTX总量达到1．2g时呼吸困难消失，而超过一半的患者在CTX总量达到2．8g时才出现吞咽困难（47例，60．3％）与肢体无力（44例，56．4％）的恢复，3组症状改善时需要的CTX总量的差异比较有统计学意义（P=0．000）。（4）CTX组在治疗期间并发肺部感染17例（21．8％），对照组并发肺部感染53例（67．9％），两组肺部感染的发生率比较差异有统计学意义（P=0．000）。（5）CTX组患者所出现的不良反应短暂且轻微。结论（1）中剂量CTX联合甲泼尼龙治疗重症肌无力危象是有效且安全的。（2）与甲泼尼龙联用，当CTX总量达到1．6g时，90％的重症肌无力危象患者能成功脱机。
Objective To evaluate the efficacy and safety of middle-dose cyclophosphamide plus methylprednisolone for myasthenia gravis （MG） patients in crisis. Methods For this prospective, open, parallel, randomized controlled trial, we recruited a total of 156 MG patients in crisis from January 1999 to October 2011 at Department of Neurology, First Affiliated Hospital, Sun Yat-sen University. They were divided into two groups of cyclophosphamide and control （ n = 78 each ）. In the cyclophosphamide group, each received methylprednisolone 500 mg/d for 3 days, then tapered to 250 mg/d and tapered half every 3 days until 62. 5 mg/d. Afterward an oral dose of prednisone was prescribed at 30 mg/d until the end of the trial. At the same time, an intravenous injection of cyclophosphamide was offered at 0. 4 g/d for 3 days and then 0.4 g/d every 3 days. In the control group, each received methylprednisolone alone. And the efficacies were assessed by absolute and relative MG scores. Results （ 1 ） There were 54 （ 69. 2% ） patients off- ventilation in 3 days in the cyclophosphamide group versus 36 （46. 2% ） patients in 8 - 14 days in the control group. Notable statistical significance existed between two groups （P = 0. 000）. （2） More than half of the patients in eyclophosphamide group with extremity weakness （ n = 44, 56% ） and dysphagia （ n = 47, 60. 3% ） significantly improved in 10 - 14 days versus 28 days in the control group. Notable statisticalsignificance existed between two groups （ P = 0. 000 ） . （ 3 ） In the cyclophosphamide group, dyspnea disappeared in 54 （69. 2% ） patients when the dose reached 1.2 g. The recovery of dysphagia （ n =47, 60. 3% ） and extremity weakness （ n =44, 56. 4% ） occurred in more than half of the patients when the dose reached 2. 8 g. Notable statistical significance existed among three groups （P = 0. 000）. （4） During the treatment period, there were 17 eases （21.8%） with pulmonary infection in the cyclophosphamide group versus 53 eases （67.9%） in the control group. Notable statistical signifieance existed between two groups （P =0. 000）. （5） Brief and minor side effects appeared in the patients of the eyelophosphamide group. Conclusion （ 1 ） The combined treatment of middle-dose cyclophosphamide and methylrednisolone for MG patients in crisis is both effective and safe. （2） When combined with methylrednisolone, 90% of patients with MG crisis are successfully off-ventilation when the dose of cyclophosphamide reaches 1.6 g.
出处
《中华医学杂志》
CAS
CSCD
北大核心
2012年第35期2473-2476,共4页
National Medical Journal of China
基金
基金项目：国家自然科学基金（201181071002）
中山大学临床医学研究5010计划项目（20112010003）
关键词
重症肌无力危象
环磷酰胺
甲泼尼龙
治疗
Myasthenia gravis crisis
Cyclophosphamide
Methylrednisolone
Treatment
