Analysis reveals benefits of immune therapy for JMG; more data needed

Treatments that suppress or alter immune system activity may help some children and adolescents with juvenile myasthenia gravis (JMG), but more studies are needed to help define the potential benefits and determine best practices.

That’s according to a meta-analysis of published studies reporting outcomes of JMG patients who were treated with immunosuppressants and immunomodulators. The included studies investigated several medications and therapeutic protocols. While many therapies showed signs of efficacy, variability between studies makes it difficult to draw clear conclusions.

“This meta-analysis could not determine a uniform effective regimen, which may rely on clinicians’ clinical experience, highlighting the need for individualized treatment for patients,” the researchers wrote.

The study, “Efficacy and safety of immunosuppressants and immunomodulators in juvenile myasthenia gravis: a systematic review and meta-analysis,” was published in the Journal of Translational Medicine.

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Addressing the clinical data gap in JMG

Myasthenia gravis (MG) is an autoimmune condition characterized by antibodies that disrupt communication between nerves and muscles, leading to fatigue and muscle weakness.

Many available treatment options aim to stop underlying autoimmune attacks. Immunosuppressants tamp down immune activity, while immunomodulators tend to regulate or adjust the immune system in more complex ways. These medication categories often overlap.

These therapies are common parts of adult MG care, but little data is available about their use in JMG, a distinct form of the disease that starts in childhood or adolescence. “The gap in clinical evidence highlights the urgency of conducting a meta-analysis to integrate existing data on immunosuppressants and immunomodulators in JMG,” the researchers wrote.

To this end, a team of researchers in China conducted a systematic review of published studies up to July 2025 that reported outcomes for children and adolescents with JMG who received immunosuppressants or immunomodulators.

A total of 24 studies, most often conducted in Asian populations, were eligible and included in the meta-analysis. The higher proportion of Asian studies may be explained by the fact that JMG rates are higher in many regions of Asia than in Europe and the Americas.

Seven of the included studies assessed glucocorticoids, the most commonly used class of corticosteroids (a type of powerful anti-inflammatory therapy), in 348 participants. Studies tested them with or without the MG-targeted therapy pyridostigmine (sold as Mestinon and generics).

Generally, patients with weakness confined to their eye-related muscles (ocular MG) benefited more from glucocorticoids than those with more widespread weakness (generalized MG).

Across the glucocorticoid studies, 12.07% of children experienced adverse events such as obesity, high blood sugar, high blood pressure, and bone problems. These are known concerns associated with glucocorticoids.

“These findings highlighted both the therapeutic value and the well-recognized burden of [glucocorticoids-associated] side effects in children,” the team wrote.

Because of the known side effects of corticosteroids, many doctors prescribe the immunosuppressant tacrolimus off-label, which may allow patients to reduce their corticosteroid dosage.

Data from nine tacrolimus studies involving 310 participants showed a pooled response rate of 86.2%. Many children experienced improvements in motor function and significant reductions in corticosteroid dosage.

“This study tends to support that tacrolimus leads to a decrease in corticosteroid dosage, though additional studies [are] needed to confirm these findings,” the researchers wrote.

While 14.2% of children in these studies experienced adverse events, they were mostly mild and rarely led to discontinuation of tacrolimus. The most common concerns were low magnesium levels and kidney problems.

Assessing antibody therapies and IVIG

Six studies, including 67 participants, tested rituximab or Soliris (eculizumab), antibody-based therapies that affect immune function. Soliris has specific regulatory approval for MG, while rituximab is used off-label.

When analyzing rituximab and Soliris studies together, the team found that the pooled response rate was 99.3% after a year. The pooled rate of adverse events was high (40.3%). These events were primarily manageable reactions to the medication infusions, such as allergic reactions and rashes.

Finally, the researchers examined four studies of intravenous immunoglobulin (IVIG) involving 84 participants. IVIG is a procedure that provides an into-the-vein infusion of antibodies from healthy donors, which is thought to modulate immune activity and neutralize self-reactive antibodies.

These studies used different IVIG protocols and dosages, making it difficult to draw broad conclusions. The response rate ranged from 47% to 94%. One-quarter of the children experienced adverse events, with the most common being mild fever and headache.

All of the included immunosuppressant or immunomodulating therapies demonstrated at least some efficacy. In particular, antibody-based therapies had a higher success rate with a relatively manageable safety profile.

Still, the team noted substantial variability in research methodologies and treatment protocols across the included studies.

Additionally, “current studies primarily focus on the efficacy of individual drugs, without direct comparison of therapeutic effects between different types of immunosuppressants/immunomodulators,” the researchers wrote. “Future research should prioritize comparative efficacy analyses of various treatment regimens.”