International Experts Update CAR T-Cell Therapy Recommendations for Autoimmune Diseases

An international panel of 31 experts has published updated consensus recommendations for the use of chimeric antigen receptor (CAR) T-cell therapy in patients with severe, treatment-refractory autoimmune diseases. The recommendations, published Oct. 5 in The Lancet Rheumatology, update guidance first issued in 2024 as CAR T-cell therapy expands beyond oncology.

The panel includes experts from the European Society for Blood and Marrow Transplantation (EBMT), the International Society for Cell & Gene Therapy and JACIE. Representatives of European rheumatology and multiple sclerosis organizations also contributed.

The recommendations cover rheumatological, neurological, hematological and pediatric indications. The guidance recommends considering CAR T-cell therapy for patients whose severe autoimmune disease remains active or progressive despite appropriate standard treatment. They advise that whenever possible, treatment should take place within a clinical trial. When a trial is unavailable, decisions should follow a documented multidisciplinary assessment involving autoimmune disease specialists and hematology or cellular therapy experts.

The recommendations also address diagnostic assessment, treatment planning, immune monitoring, management of treatment-related toxicities and long-term follow-up. They emphasize distinguishing inflammatory complications associated with CAR T-cell therapy from progression or flare of the underlying autoimmune disease. The panel recommends reporting treated patients to the EBMT Registry to support the collection of long-term data on outcomes, durability of response and late toxicities.

Although early clinical experience has shown promising results in some patients who have not responded to existing therapies, the panel notes that high-level evidence is still emerging and that evidence in children and adolescents remains limited. The recommendations are intended to evolve as new evidence and clinical experience emerge.