Department of Neurology, Kansai Medical University, 2-5-1, Shinmachi, Hirakata, Osaka 5731010, Japan.
Department of Neurology, Kansai Medical University, 2-5-1, Shinmachi, Hirakata, Osaka 5731010, Japan.
Neuromuscul Disord. 2021 Jun;31(6):512-518. doi: 10.1016/j.nmd.2021.02.010. Epub 2021 Feb 23.
The objective was to evaluate the long-term efficacy and safety of tacrolimus monotherapy in myasthenia gravis (MG) patients. Immunosuppressive drug-naïve MG patients were administered tacrolimus, followed by thymectomy in some of the cases according to the clinical guideline for MG. Additional aggressive immunosuppressive therapies were allowed if the patients without thymectomy did not achieve minimal manifestation (MM) or better status after 3 weeks of tacrolimus administration or in the thymectomized patients by 1-2 weeks after the operation (i.e., 1st evaluation). Of all 14 patients included in this study, 8 of them (57%) achieved MM or better status at the 1st evaluation, and the remaining 6 (43%), who had failed to gain MM or better status at the 1st evaluation, also achieved MM or better status with 1 course of aggressive immunosuppressive therapy. The quantitative MG (QMG) scores, MG-Activities of Daily Living (ADL) scales, and anti-acetylcholine receptor (AchR) antibody levels were significantly decreased at 6 months and maintained thereafter. At the end of the follow-up period (41-70 months), all patients were in MM or better status. None of the patients experienced severe adverse effects. Our small preliminary study indicates that long-term tacrolimus monotherapy is possibly effective and safe for MG patients.
目的是评估他克莫司单药治疗重症肌无力(MG)患者的长期疗效和安全性。根据 MG 的临床指南,给免疫抑制药物初治的 MG 患者服用他克莫司,一些病例还进行了胸腺切除术。如果未行胸腺切除术的患者在服用他克莫司 3 周后或行胸腺切除术的患者在术后 1-2 周(即第 1 次评估时)未达到最小症状(MM)或更好状态,则允许进行额外的强化免疫抑制治疗。在本研究纳入的 14 例患者中,有 8 例(57%)在第 1 次评估时达到 MM 或更好状态,其余 6 例(43%)在第 1 次评估时未达到 MM 或更好状态,但经 1 个疗程强化免疫抑制治疗后也达到 MM 或更好状态。定量重症肌无力(QMG)评分、重症肌无力日常生活活动(MG-ADL)量表和抗乙酰胆碱受体(AchR)抗体水平在 6 个月时显著下降,并在此后保持稳定。在随访期末(41-70 个月),所有患者均处于 MM 或更好状态。所有患者均未发生严重不良反应。我们的初步小样本研究表明,长期他克莫司单药治疗可能对 MG 患者有效且安全。