Safety and efficacy of CAR-T cell therapy in patients with autoimmune diseases: a systematic review

Rheumatol Int. 2025 Jan 4;45(1):18. doi: 10.1007/s00296-024-05772-5.

Abstract

Chimeric antigen receptor T-cell (CAR-T) therapy has revolutionized the treatment of various hematological malignancies. Recently, CAR-T has been used in refractory auto-immune diseases with initial encouraging results. In this systematic review, we examined the safety and efficacy of CAR-T in patients with refractory auto-immune diseases. PubMed/Medline, EMBASE, Web of Science, and Scopus search revealed 1552 articles, of which 24 were included for the final analysis. 80 patients with autoimmune diseases received CAR-T cell therapy, of which 52 patients had systemic lupus erythematosus, 16 patients had systemic sclerosis, 7 patients had idiopathic inflammatory myopathies, 2 patient had anti-phospholipid antibody syndrome, 2 patients had rheumatoid arthritis, and 1 patient had Sjogren's disease. 44 patients got CD-19 CAR-T and 36 patients got BCMA/CD-19 compound CAR-T. All the patients achieved an immunosuppression-free state at the last follow-up. Of the 47 patients with follow-up data, 79 patients developed cytokine release syndrome (CRS) and 4 patients developed neurotoxicity. None of the patients had fatal adverse events with CAR-T cell therapy. CAR-T appears to be safe and effective in patients with refractory autoimmune diseases. Future studies are crucial to further validate these findings, explore long-term outcomes, and refine the treatment protocols to enhance efficacy and safety.

Keywords: Autoimmune disorders; CAR-T Cell therapy; Cytokine release syndrome; Hematologic malignancies; Systematic review.

Publication types

  • Systematic Review
  • Review

MeSH terms

  • Adult
  • Autoimmune Diseases* / immunology
  • Autoimmune Diseases* / therapy
  • Cytokine Release Syndrome / etiology
  • Cytokine Release Syndrome / therapy
  • Female
  • Humans
  • Immunotherapy, Adoptive* / adverse effects
  • Immunotherapy, Adoptive* / methods
  • Male
  • Middle Aged
  • Receptors, Chimeric Antigen* / immunology
  • Treatment Outcome

Substances

  • Receptors, Chimeric Antigen