他克莫司治疗难治性重症肌无力的短期疗效观察

Short-Term Efficacy of Tacrolimus in Treatment of Refractory Myasthenia Gravis

目的:观察不同剂量他克莫司治疗难治性重症肌无力患者的短期疗效。方法:纳入32例难治性重症肌无力患者,分为A(2 mg/d)、B(3 mg/d)两组进行治疗,分析第2、4、12周疗效,并记录不良反应。结果:25例患者完成试验,A组11例,B组14例。与治疗前相比,A组在治疗4、12周时MG临床评分均显著降低(P<0.05);B组在治疗2、4、12周时临床评分较治疗前差异有统计学意义(P<0.05)。治疗2周时B组MG临床评分降低程度高于A组(P<0.05);4周、12周时2组比较差异无统计学意义(P>0.05);治疗12周时A、B组临床总有效率分别为81.82%、85.71%,差异无统计学意义(P>0.05)。服用他克莫司后共6例患者发生不同程度的全身/关节疼痛、恶心、腹胀、腹泻等不良反应,均经对症处理或停药后好转。2组总不良反应发生率差异无统计学意义(P=1.000)。结论:小剂量他克莫司短期内治疗难治性重症肌无力可明显改善临床症状,且不良反应发生率较低。

Objective: To observe the short-term effects of different doses of tacrolimus (FK506) in the treatment of patients with refractory myasthenia gravis. Methods: Thirty-two patients with refractory myasthenia gravis (MG) were randomly divided into two groups: group A (2 mg/d) and group B (3 mg/d). Therapeutic efficacy was analyzed at the 2nd, 4th, and 12th week, and adverse reactions were recorded. Results: Twenty-five patients completed the trial, 11 in group A and 14 in group B. Compared with before treatment, clinical scores of MG in group A were significantly decreased at 4 and 12 weeks after treatment (P<0.05). Compared with before treatment, clinical scores in group B showed significant difference at 2, 4, and 12 weeks after treatment (P<0.05). At 2 weeks after treatment, reduction in clinical scores of group B was greater than that of group A (P<0.05). There was no significant difference between the two groups at 4 and 12 weeks (P>0.05). At 12 weeks after treatment, total effective rate of group A and group B was 81.82% and 85.71%, respectively, and the difference was not statistically significant (P>0.05). After taking FK506, a total of 6 patients experienced different degrees of systemic/joint pain, nausea, bloating, diarrhea, and other adverse reactions, all of which were improved after symptomatic treatment or withdrawal. The difference in incidence of adverse reactions between the two groups was not statistically significant (P=1.000). Conclusion: Low-dose tacrolimus in the short-term treatment of refractory myasthenia gravis can significantly improve clinical symptoms, and the incidence of adverse reactions is low.