Add-on efgartigimod in myasthenic crisis: a promising treatment option

Background In myasthenia crisis (MC), plasma exchange (PE) and intravenous immunoglobulin (IVIG) are confirmed effective treatment options, but PE may not be available in time, and the response rate to IVIG is not always satisfactory. This study aimed to investigate whether addition of efgartigimod confers benefits in patients with MC. Methods This real-world retrospective pilot study examined MC patients admitted to the First Affiliated Hospital of Sun Yat-sen University between August 2023 and May 2024, who were categorized into two groups: the traditional immunotherapy group ( n = 14) and the add-on efgartigimod group ( n = 9). In the efgartigimod group, patients received 20 mg/kg efgartigimod ( n = 9), administered on the first and fifth days. Results Totally 23 patients were recruited in the study. Total hospital stays, MV duration, and non-invasive ventilation time were shorter in the efgartigimod group compared with the traditional immunotherapy group; however, these differences were not statistically significant ( p > 0.05). We subsequently applied a generalized estimating equation (GEE) to the patients over an 8-week period and observed a reduction in Myasthenia Gravis Activities of Daily Living (MG-ADL) scores in both groups; however, no significant differences were found within the groups ( p > 0.05). Survival analysis was also conducted, revealing that the addition of efgrtigimod allowed patients to reach the MSE state more quickly, which is statistically significant ( p = 0.0499). Additionally, three patients with MC who only received high-dose efgartigimod treatment all achieved favorable treatment outcomes. Conclusion This suggests that efgartigimod may serve as an alternative option for MC patients or as a rescue treatment option for refractory individuals.