Clinical effect of tacrolimus in the treatment of myasthenia gravis in children
LI Jiu-Wei, FANG Fang, REN Xiao-Tun, ZHANG Wei-Hua, YANG Xin-Ying, REN Chang-Hong, GONG Shuai, LYU Jun-Lan, WANG Xiao-Hui, WANG Xu, WU Hu-Sheng, DING Chang-Hong
Department of Neurology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing 100045, China
Abstract Objective To evaluate the efficacy and safety of tacrolimus in the treatment of children with myasthenia gravis (MG). Methods A total of 28 children with MG were treated with tacrolimus. MG-Activities of Daily Living (MG-ADL) scale was used to assess clinical outcome and safety after 1, 3, 6, 9, and 12 months of treatment. Results After tacrolimus treatment, the MG-ADL score at 1, 3, 6, 9 and 12 months was lower than that at baseline (P < 0.05), and the MG-ADL score showed a gradually decreasing trend. The response rates to tacrolimus treatment at 1, 3, 6, 9, and 12 months were 59%, 81%, 84%, 88%, and 88% respectively. At 6, 9, 12, and 18 months of treatment, 4, 13, 14, and 15 children respectively were withdrawn from prednisone. No recurrence was observed during treatment. Major adverse reactions/events were asymptomatic reduction in blood magnesium in 5 children and positive urine occult blood in 1 child, which turned negative without special treatment, and tacrolimus was not stopped due to such adverse reactions/events. One child was withdrawn from tacrolimus due to recurrent vomiting. According to CYP3A5 genotypes, all of the patients were divided into two groups:slow metabolic type (n=19) and non-slow metabolic type (fast metabolic type + intermediate type; n=9). The non-slow metabolism group received a higher dose of tacrolimus, but had a lower trough concentration of tacrolimus than the slow metabolism group (P < 0.05). The slow metabolism group had a higher response rates to tacrolimus treatment than the non-slow metabolism group (P < 0.05). Conclusions Tacrolimus appears to be effective and safe in the treatment of children with MG and is thus an option for immunosuppressive therapy. CYP3A5 genotyping has a certain guiding significance for determining the dosage of tacrolimus.
LI Jiu-Wei,FANG Fang,REN Xiao-Tun et al. Clinical effect of tacrolimus in the treatment of myasthenia gravis in children[J]. CJCP, 2020, 22(9): 964-969.
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