ACR Research Highlights Pediatric Treatment Gaps, Criteria Performance, and Early Promise of CAR T-Cell Therapy

Studies explore a variety of innovative treatments to address the need for additional care options to treat pediatric rheumatic disease patients and help close the gap of limited approved treatment for these patients.

ORLANDO –New research presented at ACR Convergence 2026 examines pediatric patient’s symptoms and potential therapies that can assist children and adolescents with symptom control, as research, medication and therapies are currently limited.

Collectively, these studies find that there continues to be limited effectiveness of currently available therapies for pediatric patients with a rheumatological condition and that these groups of patients would benefit from additional treatment options, both FDA-approved medication and the need to expand and approve therapies for more severe cases.

In study number one, Addressing Unmet Needs in Childhood-onset Systemic Lupus Erythematosus Using the Childhood Arthritis and Rheumatology Research Alliance Registry (Abstract 2426793), researchers investigate the major unmet therapeutic need for additional approved therapies for childhood-onset systemic lupus erythematosus (cSLE) due to this condition’s aggressive disease course. Belimumab is the only FDA-approved medication for cSLE; only recently approved in 2019. Analyses included 1,256 patients in the CARRA Registry fulfilling eligibility, across >70 North American sites from 2017 to March 2025. Findings show nearly all patients (99.7%) received ≥1 medication not approved for cSLE.

“This research underscores the limited effectiveness of currently available therapies for children with childhood-onset systemic lupus erythematosus as almost all children evaluated in the CARRA Registry had substantial unmet therapeutic needs. There is a continued urgent need for expanded regulatory approval of therapies for cSLE,” explains Ekemini A. Ogbu, MD, MSc, Director of Neuroinflammatory Disease Services in Rheumatology at Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.

In a second study, Classification of Pediatric APS: A Multicenter Evaluation of the 2006 and 2023 Criteria (Abstract 2452159), evaluates antiphospholipid syndrome (APS), which is associated with substantial morbidity in children, and the performance of the 2023 and 2006 APS classification criteria in a multicenter cohort of pediatric patients diagnosed with APS (n=168) and SLE or other systemic autoimmune disease (n=206). The findings indicate in pediatric APS, the strengths of the 2006 criteria included higher sensitivity and overall classification performance, and the 2023 criteria better captured non-thrombotic manifestations.

The final study, Anti-CD19 CAR T-cell therapy for the treatment of severe/refractory pediatric autoimmune diseases: a single-center case series (Abstract 2456181), identifies anti-CD19 CAR T-cell therapy as a promising treatment option in patients with severe and/or refractory autoimmune diseases (AD).

Updated since abstract submission to include two additional patients, the data presented cover eight patients – four with childhood-onset systemic lupus erythematosus (cSLE), three with juvenile dermatomyositis (JDM) and one with juvenile systemic sclerosis (jSSc) – who received a single infusion of autologous anti-CD19 CAR T cells, after which all immunosuppressants were discontinued. At last follow-up, five patients (3 of 4 cSLE and 2 of 3 JDM) were in drug-free remission, while the remaining three continued to improve off therapy. cSLE patients showed complement normalization and persistent ANA and anti-dsDNA negativity; all JDM patients achieved a major clinical response; the jSSc patient’s modified Rodnan skin score decreased from 28 to 9 at month 12. Safety was reassuring, and no flares have been recorded so far.

“These results are encouraging for children with autoimmune diseases that do not respond to conventional treatments. After a single CD19 CAR T-cell infusion, most of our patients achieved drug-free remission, and the treatment proved feasible, effective and safe. Long-term follow-up will be essential to define its future role in pediatric autoimmune diseases,” states Rebecca Nicolai, MD, Pediatric Rheumatologist at the Division of Rheumatology, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy.

These abstracts will be presented at ACR Convergence 2026. Presentation dates, times and session details are available through the official meeting program.

Media Contact
Teri Arnold
Director, Public Relations & Communications
757-272-7002
tarnold@rheumatology.org

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About the American College of Rheumatology
Founded in 1934, the American College of Rheumatology (ACR) is a not-for-profit, professional association committed to advancing the specialty of rheumatology that serves nearly 10,500 physicians, health professionals, researchers and scientists worldwide. In doing so, the ACR offers education, research, advocacy and practice management support to help its members continue their innovative work and provide quality patient care. Rheumatology professionals are experts in the diagnosis, management and treatment of more than 100 different types of arthritis and rheumatic diseases.

About ACR Convergence
ACR Convergence, the annual meeting of the American College of Rheumatology, is where rheumatology meets to collaborate, celebrate, congregate, and learn. With hundreds of sessions and thousands of abstracts, it offers a superior combination of basic science, clinical science, business education and interactive discussions to improve patient care and advance the specialty. For more information about the meeting, visit the ACR Convergence page, or join the conversation on X by following the official hashtag (#ACR26).