Clinical Trials Round Up/Research Updates-Dr. Fay Off-Label Presentation

Dr. Fay Off-Label Presentation

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Dr. Alex Fay from UCSF Benioff Children’s Hospital presented on the use of off-label monoclonal antibody therapies for refractory pediatric Myasthenia Gravis (MG) at a 2024 symposium. Pediatric MG typically responds well to treatments such as IV immunoglobulin (IVIg) and steroids, with children showing a higher remission rate than adults. However, some children require ongoing treatments, like IVIg or plasma exchange, which can be burdensome.<br /><br />The talk highlighted the potential of monoclonal antibodies to reduce treatment frequency, though data on their use in children is limited and insurance coverage can be restrictive. Studies, such as a French cohort analysis, showed that Rituximab, a monoclonal antibody, offered better outcomes than conventional immunosuppressants, including the likelihood of remission and reduced need for other medications.<br /><br />Case studies illustrated these treatments. A 9-year-old boy with seronegative MG and autoinflammatory syndrome experienced remission with Rituximab, but needed a switch to Obinutuzumab after Rituximab ceased to be effective. A 7-year-old girl with similar conditions saw partial responses with Daratumumab combined with plasma exchange, requiring less frequent treatment cycles when Obinutuzumab was added. Another case involved a 14-year-old with refractory MG and CAPS who showed improvement with Efgartigimod maintenance therapy.<br /><br />The session underlined the potential benefits of monoclonal antibodies, like Daratumumab, Ravulizumab, Eculizumab, and Obinutuzumab, while noting their variable effectiveness. Feedback from attendees was encouraged via the AANEM app, and participants were reminded to claim session credits before October 2025. This research is supported by organizations like Sarepta and the NIH, indicating an ongoing effort to optimize pediatric MG management.

Keywords

pediatric Myasthenia Gravis

monoclonal antibody therapies

Rituximab

Obinutuzumab

Daratumumab

Efgartigimod

refractory MG

treatment frequency

UCSF Benioff Children’s Hospital

insurance coverage