Patient Related Outcome Measures (Oct 2023)
Impact of Ravulizumab on Patient Outcomes and Quality of Life in Generalized Myasthenia Gravis
Abstract
Carlo Antozzi,1,2 Renato Mantegazza1 1Neuroimmunology and Neuromuscular Diseases Unit, Fondazione IRCCS Istituto Neurologico C. Besta, Milan, Italy; 2Immunotherapy and Apheresis Departmental Unit, Fondazione IRCCS Istituto Neurologico C. Besta, Milan, ItalyCorrespondence: Renato Mantegazza, Neuroimmunology and Neuromuscular Diseases Unit, Fondazione IRCCS Istituto Neurologico C. Besta, Via Giovanni Celoria 11, Milan, 20133, Italy, Email [email protected]: Myasthenia gravis (MG) is an autoimmune ion channel disorder in which antibodies to different end-plate antigens impair neuromuscular transmission, ultimately leading to muscle weakness and fatigability. In about 85% of patients with MG, autoantibodies against the acetylcholine receptor (AChR) activate the complement cascade, causing damage to the neuromuscular junction. MG is a chronic disorder for which standard therapies with corticosteroids, immunosuppressive drugs, and immunomodulation with plasma exchange or intravenous immunoglobulins modify the course of the disease, but the residual burden of physical, psychological, and social disability highlights several unmet needs, among these the need for specific, targeted, and well tolerated therapies able to improve the patients’ quality of life. Complement inhibition paved the way to precision medicine in MG since, for the first time, a specific therapy targeting a crucial pathogenetic step has been designed, tested, and proven to be effective in a controlled fashion. Ravulizumab represents the first long-acting complement inhibitor approved for treatment of patients with generalized MG, able to provide rapid, complete, and sustained complement inhibition. Ravulizumab improved the MG Activity of Daily Living scale and other clinical parameters up to 26 weeks as shown by the CHAMPION MG trial, and by its open label extension, with the added value of being administered every 8 weeks. The schedule of administration is likely to improve patients’ adherence and hence their quality of life. The introduction of complement inhibition will considerably change the traditional therapeutic strategy for MG.Keywords: Myasthenia Gravis, complement, eculizumab, ravulizumab, quality of life
