Health Service Center, Kanazawa University, Kakuma-machi, Kanazawa, Ishikawa 920-1192, Japan.
J Neurol Neurosurg Psychiatry. 2011 Sep;82(9):970-7. doi: 10.1136/jnnp-2011-300148. Epub 2011 Jul 22.
To evaluate the ability of tacrolimus to reduce the corticosteroid dose in patients with myasthenia gravis (MG) and the drug's safety in a double-blind, placebo-controlled, parallel group study.
Patients being treated with oral prednisolone at doses equivalent to 10-20 mg/day, and with stable symptoms, were randomised to tacrolimus or placebo in a 28-week double-blind study. The dose of corticosteroid was tapered with the procedures specified in the protocol. The primary efficacy endpoint was the mean daily prednisolone dose given in the last 12 weeks of the study.
Eighty patients received the study drug (40 patients in each group) and were included in the full analysis set. In the full analysis set, there was no significant difference in the primary efficacy endpoint between the two groups (p = 0.078). However, some secondary analyses suggested the steroid-sparing effect of tacrolimus. Tacrolimus was well tolerated, and no safety concerns were noted.
This study suggests that tacrolimus has a potential advantage as a steroid-sparing agent in the treatment of MG patients.
NCT00309088. Name of the trial registry: FK506 Phase 3 STUDY: A STUDY for Steroid Non-Resistant MG Patients.
评估他克莫司在重症肌无力（MG）患者中减少皮质类固醇剂量的能力，并在一项双盲、安慰剂对照、平行组研究中评估其安全性。
正在接受相当于 10-20mg/天剂量的口服泼尼松龙治疗且症状稳定的患者，按 28 周双盲方案随机分配至他克莫司或安慰剂组。皮质类固醇剂量按照方案规定的程序逐渐减少。主要疗效终点为研究最后 12-14 周的平均每日泼尼松龙剂量。
80 例患者接受了研究药物（每组 40 例），并纳入全分析集。在全分析集中，两组主要疗效终点无显著差异（p=0.078）。然而，一些次要分析提示他克莫司具有类固醇节约效应。他克莫司耐受性良好，未观察到安全性问题。
本研究表明，他克莫司作为 MG 患者的类固醇节约剂具有潜在优势。
NCT00309088。试验注册名称：FK506 三期研究：一项用于类固醇抵抗型 MG 患者的研究。
