Bi Zhuajin, Li Yue, Lin Jing, Gui Mengcui, Li Zhijun, Bu Bitao
Department of Neurology, the First Affiliated Hospital of Zhengzhou University, Zhengzhou, China.
Department of Neurology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, 1095 Jiefang Avenue, Qiaokou District, Wuhan, Hubei Province, China.
Neurol Sci. 2025 Feb;46(2):943-949. doi: 10.1007/s10072-024-07819-8. Epub 2024 Nov 6.
To evaluate the long-term efficacy and safety of tacrolimus in patients with muscle-specific kinase antibody-positive myasthenia gravis (MuSK-MG).
We performed a retrospective, single-center, and cross-sectional study analyzing medical records of 18 MuSK-MG patients treated with tacrolimus for more than 1 year. The efficacy and safety of tacrolimus were evaluated by modified Osserman scale, Myasthenia Gravis Foundation of America post-intervention status, prednisone dosage, quantitative MG (QMG) scores, MG-activity of daily living (MG-ADL) scores, anti-MuSK antibody titers, blood routine, and serum biochemicals.
After 4 weeks of tacrolimus treatment, there was a significant improvement in prednisone dose, QMG, and MG-ADL scores, which continued to improve over 1 year. In addition, clinical grade of modified Osserman scale was improved in all patients, 16 (88.9%) of whom were asymptomatic at the last visit. More importantly, the mean titers of anti-MuSK antibody were significantly decreased from 0.777 ± 0.381 to 0.283 ± 0.178 nmol/L after a median of 1.4 years of tacrolimus treatment in 9 patients with MuSK-MG (P = 0.015). All patients achieved minimal manifestations status (MMS) after tacrolimus treatment (range, 4-32 weeks). Subsequently, seven patients (38.9%) underwent a taper of tacrolimus dosage. However, four patients (57.1%) experienced an exacerbation. Adverse events occurred in 2 patients (11.1%), all of which were mild and resolved after the tacrolimus dose was adjusted or discontinued.
Our results suggest that tacrolimus may be an effective and safe steroid-sparing treatment for patients with MuSK-MG. However, tacrolimus should be carefully tapered to avoid disease exacerbation.
评估他克莫司在肌肉特异性激酶抗体阳性重症肌无力（MuSK-MG）患者中的长期疗效和安全性。
我们进行了一项回顾性、单中心横断面研究，分析了18例接受他克莫司治疗超过1年的MuSK-MG患者的病历。通过改良的奥斯曼量表、美国重症肌无力基金会干预后状态、泼尼松剂量、定量重症肌无力（QMG）评分、重症肌无力日常生活活动（MG-ADL）评分、抗MuSK抗体滴度、血常规和血清生化指标来评估他克莫司的疗效和安全性。
他克莫司治疗4周后，泼尼松剂量、QMG和MG-ADL评分有显著改善，并在1年中持续改善。此外，所有患者改良奥斯曼量表的临床分级均有改善，其中16例（88.9%）在最后一次随访时无症状。更重要的是，9例MuSK-MG患者在接受他克莫司治疗中位时间1.4年后，抗MuSK抗体的平均滴度从0.777±0.381显著降至0.283±0.178 nmol/L（P = 0.015）。所有患者在他克莫司治疗后均达到最小表现状态（MMS）（范围为4 - 32周）。随后，7例患者（38.9%）进行了他克莫司剂量的减量。然而，4例患者（57.1%）病情加重。2例患者（11.1%）发生不良事件，均为轻度，在调整或停用他克莫司剂量后缓解。
我们的结果表明，他克莫司可能是一种对MuSK-MG患者有效且安全的类固醇替代治疗方法。然而，应谨慎调整他克莫司剂量以避免疾病加重。
