Juvenile myasthenia gravis (MG) is a rare autoimmune disorder, with an estimated US incidence of 1.2 per million person-years. Manifestations can range from mild ocular symptoms alone to profound muscle weakness and fatigability, to life-threatening crises, requiring hospitalization, intubation and -ventilation, and intensive care with immunosuppressants. Current standard-of care treatments are associated with significant adverse events. Further, up to a quarter of patients may not respond at all to existing treatments, leading to higher reliance on emergent treatment options such as IVIG and/or plasma exchange (PLEX) to manage the disease. New, safe and more effective therapies are urgently needed, as are clinical sites with juvenile MG expertise to assess these therapies via clinical trials.
To evaluate the juvenile MG patient density accessing emergent treatments for MG in the US by zip code and their proximity to sites participating in juvenile MG trials
A total of 27 sites participating in ongoing juvenile MG trials was identified via the website Clinicaltrials.gov accessed on December 15, 2021. Using US Real World Data, regions with a high density of juvenile MG patients with pre-selected characteristics (i.e. age, ICD-10-CM code, diagnosed between January 2015 through December 2021, and at least 1 visit for IVIG and/or PLEX treatment) were identified. Heatmaps were created for this cohort and overlaid with the 27 sites participating in juvenile MG trials. Several regions with high heat maps had no nearby trial site.
There is an urgent need for safer and more effective treatments for juvenile MG. Several new therapeutic agents are currently being tested in clinical trials, yet access to trial sites is limited for the majority of patients seen in the US, who are dependent on local physicians for their treatment and continue to receive hospital-based emergent treatments such as IVIG or PLEX for their disease management.