Abstract Presenter: Rebecca Nicolai, MD, Pediatric Rheumatologist, Division of Rheumatology, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy
Abstract Title: Anti-CD19 CAR T-Cell Therapy for the Treatment of Severe/Refractory Pediatric Autoimmune Diseases: A Single-Center Case Series
Abstract Session: Abstracts: Abstracts: Pediatric Rheumatology – Clinical: Topics in Pediatric Rheumatology (2845-2850), 1–2:30 p.m., Tuesday, November 10
What is your abstract about?
Dr. Nicolai: It reports outcomes in eight children with severe or refractory autoimmune diseases: four with lupus, three with juvenile dermatomyositis, and one with juvenile systemic sclerosis. Each received a single infusion of fresh autologous anti-CD19 chimeric antigen receptor (CAR) T cells, and all immunosuppressants were stopped afterward. Five patients reached drug-free remission, and the other three keep improving off therapy. The safety profile was reassuring, and no flares have occurred so far.
Why did you decide to investigate this topic?
Dr. Nicolai: Some children with autoimmune diseases do not respond to multiple lines of immunosuppression. They face severe organ damage and long-term toxicity from treatment and ongoing inflammation in target tissues. CAR T cells can deeply reset the immune system, and we wanted to evaluate whether the remarkable results seen in adults can be confirmed also in children.
What are you working on next related to this research?
Dr. Nicolai: Long-term follow-up of efficacy and safety, through dedicated registries, in order to better define the role of CAR T cells in pediatric autoimmune diseases. We are also working with other European centers to share data and harmonize protocols.
What excites you most about your work?
Dr. Nicolai: After a single infusion of anti-CD19 CAR T cells, children who had previously not responded to multiple immunosuppressive treatment lines are now in remission without drugs. They were able to get back to school, sports, and a normal childhood.
What are you most looking forward to at ACR Convergence 2026 in Orlando?
Dr. Nicolai: Sharing our experience with the international community and learning from other groups working on cellular therapies in autoimmunity. I also look forward to building new collaborations to bring these treatments to more children.
THERE’S STILL TIME TO JOIN US
If you haven’t registered for ACR Convergence 2026, you can still register on-site at the Orange County Convention Center in Orlando, Florida, through November 11. All registered participants receive on-demand access to scientific sessions through September 30, 2027.
