Systematic Review of Chimeric Antigen Receptor T-cell Therapy in Autoimmune Diseases

This study aimed to systematically summarize available clinical data on the use of chimeric antigen receptor (CAR) T-cell therapy in autoimmune diseases (ADs).

A systematic review was conducted using PubMed and other literature databases to identify publications on CAR-T therapy in ADs published since 2020. Eligible studies included single-case reports and case series from clinical trials, while studies involving duplicated patient populations were excluded in accordance with PRISMA guidelines. Extractable patient-level data were analyzed for key clinical outcomes.

A total of 32 studies encompassing 124 patients with ADs who received CAR-T therapy were included. The primary diseases treated were systemic lupus erythematosus (SLE) and myasthenia gravis (MG), with smaller numbers of patients reported in other ADs. In SLE trials, 83% achieved clinical remission or low disease activity following CAR-T therapy, within the first few months following infusion. In MG trials, minimal symptom expression was achieved in 73%. CAR-T disappearance and B-cell reconstitution generally occurred within the first few months following infusion and were predominantly of a naïve phenotype. Mild cytokine release syndrome was the most common adverse event; serious adverse events were reported in 4.8% of patients without treatment-related deaths.

Preliminary clinical evidence suggests that CAR-T therapy is associated with meaningful clinical response in selected ADs. Larger studies with standardized outcome measures and longer follow-up are needed to better define its safety and efficacy.