A tacrolimus course of more than a year is effective and well-tolerated in young children with myasthenia gravis (MG), according to a new study published in the Journal of Clinical Neuroscience. Moreover, the treatment reduces the disease symptoms and the dose of oral prednisone required and, therefore, associated adverse events.
To evaluate the efficacy and long-term safety of tacrolimus in children with MG as young as 2 years of age, a research team from China analyzed 21 patients. In some patients, corticosteroids were ineffective, while others could not tolerate the drug. Some patients were dependent on corticosteroids.
Read more about the treatment of MG
The results showed that at the last visit, most patients (71.4%) treated with tacrolimus achieved minimal manifestation or better.
The symptoms evaluated by MG Activities of Daily Living (MG-ADL) score improved significantly 1 month after the initiation of the treatment and continued to improve throughout the study and after. Moreover, the dose of prednisone decreased significantly after 3 months, with most patients (61.9%) being ultimately weaned off it completely.
The researchers reported that the MG-ADL score was the only clinical factor of the efficacy of tacrolimus.
Regarding side effects, 1 patient experienced intraocular pressure and transient urine microprotein.
“This study adds to the research on the efficacy and side effects of tacrolimus in the treatment of young children with MG,” the researchers said.
MG is an autoimmune disorder affecting the neuromuscular junction, causing muscle weakness and fatigue. Although the disease is uncommon in children in Europe, half of the patients with MG in China are children.
Tacrolimus is an immune suppressant often used to reduce the risk of organ rejection following transplantation. It is also used in patients with MG. However, there are currently no formal guidelines for using immunosuppressants in children with the disease.
Zhang Y, Zhang M, Zhang L, Zhou S, Li W. Long-term efficacy and safety of tacrolimus in young children with myasthenia gravis. J Clin Neurosci. Published online September 3, 2023. doi:10.1016/j.jocn.2023.08.022