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.