Objectives 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.
Methods 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.
Results 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.
Conclusions This study suggests that tacrolimus has a potential advantage as a steroid-sparing agent in the treatment of MG patients.
Clinical trial registration number NCT00309088. Name of the trial registry: FK506 Phase 3 Study: A Study for Steroid Non-Resistant MG Patients.
- Clinical trials randomised
- controlled (consort agreement)
- autoimmune disease
- myasthenia gravis
- health policy and practice
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Funding Supported by Astellas Pharma.
Competing interests All authors who participated as consultants and medical advisors to Astellas Pharma received fees as consultants of Astellas Pharma. HY served as a member of the Research Committee on Neuroimmunological Diseases of the Ministry of Health, Labour and Welfare of Japan. HY is funded by KAKENHI (19590984 and 21600002) and supported by the Research Committee on Neuroimmunological Diseases of the Ministry of Health, Labour and Welfare of Japan.
Ethics approval Ethics approval was provided by the medical facilities that participated in this study.
Provenance and peer review Not commissioned; externally peer reviewed.
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