Effective treatment of a 13-year-old boy with steroid-dependent ocular myasthenia gravis using tacrolimus.
ABSTRACT Over the past several years, tacrolimus has attracted attention as a new therapeutic drug for myasthenia gravis (MG), but few reports have considered its use for MG in pediatric patients, and most of these have focused on severe systemic MG. In this case report, we used tacrolimus to successfully treat a 13-year-old boy with ocular MG who had suffered from severe steroid complications, including a failure of thrive and osteoporosis. He first showed symptoms of ocular MG at age 2years 3months. At age 13years, he was receiving PSL (3.75mg/day), but the symptoms of ocular MG recurred. We increased the dosage of oral PSL up to 30mg/day, and three courses of mPSL pulse therapy were applied, but these therapies had only limited effect, and his symptoms worsened. Tacrolimus was started at 0.4mg/day (0.011mg/kg/day), and every 2weeks the dose was gradually increased by 0.2mg/day. His symptoms of MG began to improve 3weeks after the initial administration of tacrolimus. Approximately 3months after the start of tacrolimus administration, PSL was discontinued. Currently, at 1year and 4months after the start of tacrolimus administration, while slight ptosis is observed in the evening, it does not influence his daily life, and his condition remains comparable to that when he stopped taking PSL. No adverse effects of tacrolimus have been recognized. In pediatric patients with steroid-dependent ocular MG without thymectomy, tacrolimus may be a safe and effective alternative to steroid and thymectomy.