Car-T Cell Treatment in Refractory Autoimmune Disease

A case series provides evidence supporting the long-term safety and efficacy of autologous CD19 CAR-T cell treatment in systemic lupus erythematosus (SLE), idiopathic inflammatory myopathies (IIM), and systemic sclerosis (SSc), researchers reported in Annals of the Rheumatic Diseases.

From February 2021 to May 2023, 15 patients (8 with SLE, 4 with SSc, and 3 with IIM) were treated with CD19 CAR-T cells. The cohort consisted of 10 women and 5 men with a median age of 36 years and a median disease duration of 3 years. All patients had previously failed multiple immunosuppressive treatments.

Safety parameters, including cytokine-release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), myelotoxicity, and infections, were monitored for up to 3 years post-infusion. Efficacy was assessed using various clinical response criteria up to three years after CAR-T cell infusion.

The median follow-up time after infusion was 18 months, with some patients followed for more than 2 years. Safety results showed no high-grade CRS; 8 patients experienced grade 1 CRS, and 1 experienced grade 2 CRS. There was only a single case of grade 1 ICANS. No acute myelotoxicity occurred, but 1 patient developed late-stage grade 4 neutropenia, which resolved with treatment. Severe infections were rare, with only 1 case of pneumonia requiring hospitalization.

All patients with SLE achieved remission, according to the Definition of Remission in SLE (DORIS); all patients with IIM reached a major response per ACR/EULAR criteria; and all patients with SSc showed improvement on the EUSTAR activity index. Immunosuppressive therapy, including glucocorticoids, was discontinued in all patients.

The researchers concluded that CAR-T therapy for autoimmune illnesses warrants further clinical study.

Reference
Taubmann J, Müller F, Fagni F, et al. OP0027 Long-term safety and efficacy of car-t cell treatment in severe and treatment refractory autoimmune disease. Annals of the Rheumatic Diseases 2024;83:137.