Introduction
Myasthenia gravis (MG) is a rare, chronic autoimmune disorder affecting the neuromuscular junction (NMJ).1 MG is characterized by muscle fatigability and weakness, in most cases, initially only affecting the eye muscle but progressing to generalized MG (gMG) involving the muscles of the head, neck, trunk, limbs, and/or respiratory system in roughly 80% of patients approximately 2-3 years following disease onset.2 Myasthenic crisis is a common complication of MG, affecting 15-20% of patients, and is characterized by respiratory failure requiring mechanical ventilation and intensive care and is often associated with substantial long-term physical and mental consequences.2,3 The burden of MG is considerable, negatively impacting patients’ daily activities; quality of life (QoL); and emotional, social, and economic well-being.3 Furthermore, the current MG treatment algorithm, particularly long-term treatment with corticosteroids or nonsteroidal/nonspecific immunosuppressive therapies, have known serious, often troublesome, side effects and unpredictable treatment responses that lead to patient dissatisfaction and reduced treatment adherence, further complicating MG management.1,3
Targeted approaches to gMG management, namely eculizumab (Soliris®) and ravulizumab (Ultomiris®), were approved in the United States and Europe in 2017 and 2022,4,5 respectively, with data from two open-label extension studies pointing to the long-term safety and sustained effectiveness of both medicines.2,6Eculizumab is a humanized monoclonal antibody that binds specifically and with high affinity to the human terminal complement protein C5, preventing destruction of the NMJ and consequent muscle weakness and fatigability.3 Ravulizumab is also a C5 inhibitor, engineered from eculizumab to have a longer half-life, maintaining therapeutic serum concentrations over an 8-week dosing interval (versus a 2-week interval with eculizumab).5
Despite the clinical benefits of these more targeted treatments, given the chronic nature of gMG, the likelihood of their success in disease management in the real world is multifactorial. Confirmation of their long-term effectiveness and safety in routine clinical practice and strategies for overcoming challenges associated with their accessibility (e.g., high cost and limited availability) are needed. We report a patient with 20 years of gMG, who after 16 years of refractory disease achieved and, importantly, has maintained a stable condition for the last 3 years with eculizumab/ravulizumab therapy.