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.