Addressing Treatment Gaps in Adolescents With Myasthenia Gravis: Jonathan Strober, MD

WATCH TIME: 3 minutes

"It’s really going to be amazing to have these drugs tested in this population and also confirm that the dosing we’re using is the proper dose for this patient population. We always use adult-based dosing, and it’s, we’re not sure if it’s the right one for pediatrics or not."

In recent years, myasthenia gravis (MG) has been among one of the most promising neurologic disease conditions to treat, with a handful of new approved therapies available those living with the disease. While the expansion in medications has been encouraging, the drugs are only indicated for adults living with the disease, the population that was tested in clinical trials. One investigational agent, nipocalimab (Johnson & Johnson) was recently tested in a phase 2/3 study, dubbed VIBRANCE-MG (NCT), which features adolescents with MG.

Data from the small-scale study, which featured 7 adolescents aged 12-18 with seropositive MG, was recently presented at the 2024 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) meeting, held October 15-18, in Savannah, Georgia. Of the included sample, 5 completed the 24-week treatment period. Overall, nipocalimab treatment resulted in statistically significant reduction in total serum immunoglobulin (IgG), the primary end point, at week 24, with a mean percent change of –68.98% (SD, 7.561).

Shortly after the meeting, lead investigator Jonathan Strober, MD, director of Clinical Services for Child Neurology and director of the Muscular Dystrophy Clinic at UCSF Benioff Children’s Hospital, sat down to discuss the findings. In the conversation, Strober provided background on the origins of the study and the importance behind studying adolescents with MG, a patient population often left out of clinical trials. Strober also discussed the complexities of diagnosing MG in adolescents, especially with the presence of seronegative patients who exhibit symptoms without detectable antibodies. Overall, he stressed that the study offers hope for more targeted treatment options that consider the specific needs and challenges of younger patients with MG.

Click here for more AANEM 2024 coverage.

REFERENCES
1. Strober J, Black S, Ramchandren S, et al. Safety and effectiveness of nipocalimab in adolescent participants in the open label phase 2/3 Vibrance-MG clinical study. Presented at: 2024 AANEM Annual Meeting; October 15-18; Savannah, GA.