How effective is tacrolimus therapy in juvenile myasthenia gravis?

How effective is tacrolimus therapy in juvenile myasthenia gravis? | Image credit: Contemporary Pediatrics

Tacrolimus (TAC) improves symptoms in most cases of juvenile myasthenia gravis (JMG), with few serious adverse drug reactions (ADRs), according to a meta-analysis of 9 trials involving retrospective/prospective comparison studies in 313 individuals younger than 18 years with JMG. Two studies compared TAC and corticosteroids (CSs), and the remaining 7 had a single-group pretest-posttest design.

In the 6 trials that reported myasthenia gravis activities of daily living scores, TAC had a significant effect in reducing these scores. These improvements were observed as early as 1 month after initiating TAC treatment. Further, the degree of improvement increased along with an increase in follow-up duration. TAC also had a positive significant effect in reducing quantitative myasthenia gravis scores in the 3 trials that considered these scores. In addition, TAC was associated with reducing serum acetylcholine reception titers, according to data from the 3 studies. Finally, TAC therapy was associated with a reduction in CS dose.

A total of 23 ADRs related to TAC were reported in 4 studies; none were serious. However, 2 studies reported that TAC-related adverse effects led 13 patients to discontinue participation in the study.

Investigators concluded that TAC may be a beneficial treatment option for patients with JMG.

THOUGHTS FROM DR FARBER

TAC looks to be a useful second-line addition for myasthenia gravis. My suspicion, however, is that it will soon become a third-line medicine, as I expect monoclonal antibodies to become first-line treatment in pediatrics in the next few years.

Reference

Jiang A, Hu Q, Wang Z, Wu F. Efficacy and safety of tacrolimus therapy in patients with juvenile myasthenia gravis: a single-arm meta-analysis. Pediatr Neurol. 2025;166:32-38. doi:10.1016/j.pediatrneurol.2025.02.007