Discussion and conclusion
Case reports highlighting eculizumab and ravulizumab as effective rescue therapy for MG are increasing, with emerging evidence supporting their positive impact on the disease’s long-term progression.7 The current case report confirms the long-term effectiveness and safety of eculizumab and ravulizumab as a rescue treatment for gMG in a patient who was unresponsive to conventional immunosuppressive therapies for 16 years. For this patient, eculizumab/ravulizumab treatment was a “game changer” in gMG management, not only because of a rapid improvement in clinical outcomes but also the fact that these targeted treatments have maintained efficacy over the last 3 years, with a significant positive impact on the patient’s QoL.
Similar to a previous report,8 discontinuing eculizumab resulted in significant worsening of the patient’s condition. Considering discontinuation occurred due to accessibility issues, developing adequate strategies to support the accessibility and long-term use of targeted immunotherapy in life-long conditions such as gMG should be prioritized for the patient’s well-being. Favorably, it is worth noting that the patient’s condition quickly improved upon reinitiating eculizumab treatment, on par with the previous report.8 Additionally, the patient was successfully switched to ravulizumab—administered at a lower frequency and available at a lower cost—without any negative effect on clinical outcomes. This favorable response is in line with results from a recent study, where switching from eculizumab to ravulizumab was found to be effective for refractory acetylcholine receptor positive gMG patients in a clinical setting.9 The patient has been receiving ravulizumab treatment since the switch in 2022 and is doing well, with the gMG under control.
As real-world evidence accumulates, the importance of complement C5 inhibitors in gMG treatment is becoming increasingly clear, with eculizumab and ravulizumab offering promising long-term management for patients with severe clinical conditions and refractory disease.