{"title":"他克莫司治疗抗MuSK抗体阳性肌无力的长期疗效和安全性:一项回顾性单中心队列研究。","authors":"Zhuajin Bi, Yue Li, Jing Lin, Mengcui Gui, Zhijun Li, Bitao Bu","doi":"10.1007/s10072-024-07819-8","DOIUrl":null,"url":null,"abstract":"<p><strong>Objective: </strong>To evaluate the long-term efficacy and safety of tacrolimus in patients with muscle-specific kinase antibody-positive myasthenia gravis (MuSK-MG).</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusion: </strong>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.</p>","PeriodicalId":19191,"journal":{"name":"Neurological Sciences","volume":" ","pages":""},"PeriodicalIF":2.7000,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Long-term efficacy and safety of tacrolimus in anti-MuSK antibody-positive myasthenia gravis: a retrospective single-center cohort study.\",\"authors\":\"Zhuajin Bi, Yue Li, Jing Lin, Mengcui Gui, Zhijun Li, Bitao Bu\",\"doi\":\"10.1007/s10072-024-07819-8\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Objective: </strong>To evaluate the long-term efficacy and safety of tacrolimus in patients with muscle-specific kinase antibody-positive myasthenia gravis (MuSK-MG).</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusion: </strong>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.</p>\",\"PeriodicalId\":19191,\"journal\":{\"name\":\"Neurological Sciences\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":2.7000,\"publicationDate\":\"2024-11-06\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Neurological Sciences\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1007/s10072-024-07819-8\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"CLINICAL NEUROLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Neurological Sciences","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s10072-024-07819-8","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}
Long-term efficacy and safety of tacrolimus in anti-MuSK antibody-positive myasthenia gravis: a retrospective single-center cohort study.
Objective: To evaluate the long-term efficacy and safety of tacrolimus in patients with muscle-specific kinase antibody-positive myasthenia gravis (MuSK-MG).
Methods: 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.
Results: 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.
Conclusion: 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.
期刊介绍:
Neurological Sciences is intended to provide a medium for the communication of results and ideas in the field of neuroscience. The journal welcomes contributions in both the basic and clinical aspects of the neurosciences. The official language of the journal is English. Reports are published in the form of original articles, short communications, editorials, reviews and letters to the editor. Original articles present the results of experimental or clinical studies in the neurosciences, while short communications are succinct reports permitting the rapid publication of novel results. Original contributions may be submitted for the special sections History of Neurology, Health Care and Neurological Digressions - a forum for cultural topics related to the neurosciences. The journal also publishes correspondence book reviews, meeting reports and announcements.