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Science News: Tacrolimus Improves Symptoms of Children with Myasthenia Gravis Refractory to Prednisone

Chanchan L et al. Tacrolimus Improves Symptoms of Children with Myasthenia Gravis Refractory to Prednisone. Pediatric Neurology 2017 Sept 5. 
Submitted by Leigh Maria K. Ramos-Platt, MD, News Science Editorial Board
Edited by Niranjan N. Singh, MD, News Science Editorial Board
This study evaluated the efficacy of Tacrolimus in Chinese pediatric autoimmune Myasthenia Gravis patients who were either (1) refractory to prednisone plus pyridostigmine therapy, and/or (2) having significant side effects. The dose used was 1-2 mg/kg/day to start and adjusted for response and serum level. No other immunomodulation was used. Thirteen out of 14 patients completed this study. The patients were between five and 14 years of age and were followed at Tongji Hospital in China between January 2015 and December 2016. Patients were evaluated every four months for a year in treatment. There was no placebo arm. Eleven of the 13 patients were ocular only. None of the patients underwent thymectomy. There were no serious side effects reported. Improvements were seen in the Quantitative Myasthenia score (QMG), Myasthenia Specific Manual Testing (MG-MMT), and Myasthenia Gravis activities of daily living (MD-ADL) compared to baseline. Acetylcholine Receptor antibody serum level decreased during treatment of Tacrolimus. Most importantly, prednisone/prednisolone was able to be greatly reduced with improvement of symptoms while on Tacrolimus from 26.15+/- 14.02 mg daily to 1.15 +/- 2.19 mg daily. Ten out of the 13 patients were able to come off of prednisone altogether.
Comment: While this study does have its limitations (e.g. only a single ethnic population, predominantly Ocular Mysasthenia, and a small number), it provides insight into an area that needs to be further explored. In the pediatric population, the effects of long-term high-dose chronic steroids can have a lasting impact on a patient as they enter into adulthood. This small study demonstrated that Tacrolimus may be a viable steroid sparing option in pediatric patients with Myasthenia Gravis. Further studies will be needed.

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