小剂量他克莫司在难治性重症肌无力中的应用

摘要

Objective To discuss the clinic effect and adverse reaction of low-dose tacrolimus in the treatment of refractory myasthenia gravis(MG).Methods 14 cases of refractory myasthenia gravis patients admitted from 2011 April to 2013 April were selected ,all patients took 0.1 mg · kg-1 · d-1 tacrolimus on the basis of prior treatment ,and took 20 weeks as a course of treatment. The activities of daily living scale (ADL) ,the United States of America MG Foundation (MGFA) evaluation of dis-easeseverityscale,Ossermanratingandhandmusclestrengthtable(MMT)wereusedtoevaluatetheclinicseffectoflow-dose tacrolimus on patients with refractory myasthenia gravis in the fourth week ,8th week ,16th week and 20th week of treatment process ,and the adverse reactions were detected and recorded. Results All patients were treated for 20 weeks. Compared with pretherapy ,each index of patients improved significantly after 20 weeks ,the degree of improvement in ADL was 49.5% ~100% ,the degree of improvement in MGFA was 36.5% ~89.6% ,Osserman rating decreased 2 levels ,the degree of improve-ment in MMT was 55.3% ~90.8% ,compared with pretherapy ,the difference was significant ,with statistical significance (P<0.05). In addition ,only 2 cases among 14 patients had a headache ,dizziness ,high blood pressure and other adverse reac-tions which released within one week ,and there was not any adverse reaction on other patients.Conclusion Long-term low-dose tacrolimus in the treatment of refractory myasthenia gravis was safe and effective ,but because of the small sample size , there still needs further high quality study to verify the clinical effects of the drug.%目的：探讨小剂量他克莫司治疗难治性重症肌无力（M G ）的临床效果与不良反应。方法选取2011-04-2013-04收治的难治性重症肌无力患者14例，所有患者在原来治疗的基础上服用0.1 mg · kg-1· d-1他克莫司，20周为1个疗程。采用日常生活量表（ADL）、美国MG基金会（MGFA）疾病严重程度评价量表、Osserman评级和徒手肌力量表（MMT），在患者治疗的第4周、8周、16周和20周分别评价治疗效果，检测并记录不良反应情况。结果所有患者均接受20周的疗程，20周后患者各个指标评分较治疗前均有明显改善，其中ADL改善程度为49.5％～100％，MGFA的改善程度为36.5％～89.6％， Osserman评级降低了2个级别，MMT的改善程度为55.3％～90.8％，与治疗前比较差异具有统计学意义（P＜0.05）。另外，14例患者中仅2例在2周左右时出现头痛、头晕、高血压等不良反应，1周内缓解，其他患者未出现任何不良反应。结论长期小剂量服用他克莫司治疗难治性重症肌无力安全有效，但因样本量少，仍需要进一步的研究验证该药的临床效果。