Single-cell profiling of immune reset in patients with refractory generalized myasthenia gravis receiving autologous CD19/BCMA CAR-T cell therapy - PubMed
4 hours ago
- #myasthenia gravis
- #CAR-T therapy
- #immune reset
- Autologous CD19/BCMA CAR-T cell therapy was tested in patients with refractory generalized myasthenia gravis (gMG) without prior lymphodepletion.
- The therapy showed a favorable safety profile, with minimal symptom expression achieved in five out of six patients by day 90.
- Patients discontinued glucocorticoids and reduced immunosuppressant use, with sustained responses through day 120-150.
- Immune profiling revealed transient declines in memory B cells and plasma cells, with repopulated B cells showing attenuated B cell receptor signaling.
- Clonal expansion of T and B cells was significantly reduced, indicating a systemic immune reset.
- The study suggests CD19/BCMA CAR-T cell therapy is safe and effective for refractory gMG, warranting further investigation in larger clinical trials.