Clinical effect of tacrolimus in the treatment of myasthenia gravis in children.
- Author:
Jiu-Wei LI
1
;
Fang FANG
;
Xiao-Tun REN
;
Wei-Hua ZHANG
;
Xin-Ying YANG
;
Chang-Hong REN
;
Shuai GONG
;
Jun-Lan LYU
;
Xiao-Hui WANG
;
Xu WANG
;
Hu-Sheng WU
;
Chang-Hong DING
Author Information
1. Department of Neurology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing 100045, China. 13641290689@163.com.
- Publication Type:Journal Article
- MeSH:
Activities of Daily Living;
Child;
Humans;
Immunosuppressive Agents;
Myasthenia Gravis;
drug therapy;
Neoplasm Recurrence, Local;
Tacrolimus;
therapeutic use
- From:
Chinese Journal of Contemporary Pediatrics
2020;22(9):964-969
- CountryChina
- Language:Chinese
-
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 At 6, 9, 12, and 18 months of treatment, 4, 13, 14, and 15 children respectively were withdrawn from prednisone.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 One child was withdrawn from tacrolimus due to recurrent vomiting.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.
