Long-term outcomes of anti-CD19 CAR T cell therapy in refractory myasthenia gravis: A case series
TL;DR
Data support anti-CD19 CAR T cell therapy as a durable, effective intervention for refractory MG and warrant further evaluation in prospective controlled clinical trials.
Abstract
Summary Anti-CD19 CAR T cell therapy represents an emerging therapeutic approach for generalized, treatment-refractory myasthenia gravis (MG), a predominantly B-cell-mediated autoimmune disease for which durable treatment-free remission remains an unmet clinical goal. Despite recent advances, current targeted therapies generally require lifelong repeated administration and rarely induce durable treatment-free remission. We report on three patients with severe, treatment-resistant MG, including one patient with concomitant rheumatoid arthritis, treated with autologous, fully human anti-CD19 CAR T cells. All three patients achieve rapid, sustained clinical MG remission for at least 19 months, allowing discontinuation of MG-specific immunotherapies and substantial improvement in clinical and functional outcomes despite persistent detectable anti-AChR autoantibody titers. B cell depletion is profound, and treatment-related adverse events remain transient and manageable during long-term follow-up. These data support anti-CD19 CAR T cell therapy as a durable, effective intervention for refractory MG and warrant further evaluation in prospective controlled clinical trials.