Fan Zhirong, Li Zunbo, Shen Faxiu, Zhang Xueping, Lei Lin, Su Shengyao, Lu Yan, Di Li, Wang Min, Xu Min, Da Yuwei
Department of Neurology, Xuanwu Hospital, Capital Medical University, Beijing, China.
Department of Neurology, Xi'an Gaoxin Hospital, Xi'an, China.
Front Neurol. 2020 Oct 27;11:594152. doi: 10.3389/fneur.2020.594152. eCollection 2020.
Tacrolimus (TAC) has been proven to be a rapid-acting, steroid-sparing agent for myasthenia gravis (MG) therapy. However, evidence related to the effectiveness of TAC alone is rare. Therefore, this study was performed to investigate the effect of TAC monotherapy in MG patients. Forty-four MG patients who received TAC monotherapy were retrospectively analyzed. A mixed effect model was used to analyze improvements in MG-specific activities of daily living scale (MG-ADL), quantitative MG score (QMG) and MG-ADL subscores. Kaplan-Meier analysis was used to estimate the cumulative probability of minimal manifestations (MM) or better. Adverse events (AEs) were recorded for safety analyses. Of the patients receiving TAC monotherapy, MG-ADL scores were remarkably improved at 3, 6 and 12 months compared with scores at baseline (mean difference and 95% CIs: -3.29 [-4.94, -1.64], -3.97 [-5.67, -2.27], and -4.67 [-6.48, -2.85], respectively). QMG scores significantly decreased at 6 and 12 months, with mean differences and 95% CIs of -4.67(-6.88, -2.45) and -5.77 (-7.55, -4.00), respectively. Estimated median period to achieve "MM or better" was 5.0 (95% CIs, 2.8, 7.2) months. Ocular MG (OMG) and generalized MG (GMG) showed similar therapeutic effects in cumulative probabilities of "MM or better" (-value = 0.764). A better response was observed in MG-ADL subscores for ptosis and bulbar symptoms. AEs occurred in 37.5% of patients and were generally mild and reversible. TAC monotherapy is a promising option to rapidly alleviate all symptoms of MG, especially for ptosis and bulbar symptoms.
他克莫司（TAC）已被证明是一种用于治疗重症肌无力（MG）的速效、可替代类固醇的药物。然而，关于单独使用TAC有效性的证据很少。因此，本研究旨在调查TAC单药治疗对MG患者的疗效。对44例接受TAC单药治疗的MG患者进行回顾性分析。采用混合效应模型分析MG特异性日常生活活动量表（MG-ADL）、定量MG评分（QMG）和MG-ADL子评分的改善情况。采用Kaplan-Meier分析估计最小表现（MM）或更好表现的累积概率。记录不良事件（AE）以进行安全性分析。在接受TAC单药治疗的患者中，与基线时的评分相比，MG-ADL评分在3个月、6个月和12个月时显著改善（平均差值和95%可信区间分别为：-3.29[-4.94，-1.64]、-3.97[-5.67，-2.27]和-4.67[-6.48，-2.85]）。QMG评分在6个月和12个月时显著降低，平均差值和95%可信区间分别为-4.67（-6.88，-2.45）和-5.77（-7.55，-4.00）。达到“MM或更好”的估计中位时间为5.0（95%可信区间，2.8，7.2）个月。眼肌型MG（OMG）和全身型MG（GMG）在“MM或更好”的累积概率方面显示出相似的治疗效果（P值=0.764）。在MG-ADL子评分中，上睑下垂和延髓症状有更好的反应。37.5%的患者发生AE，且一般为轻度且可逆。TAC单药治疗是迅速缓解MG所有症状的一个有前景的选择，尤其是对上睑下垂和延髓症状。
