Early results presented at ACR Convergence this week in Orlando show drug-free remission in children with severe autoimmune conditions, as new research exposes major gaps in paediatric rheumatology treatments.
CAR T-cell therapy has achieved drug-free remission in five of eight children with severe autoimmune diseases, raising hopes for a new treatment option for patients who fail to respond to conventional therapies.
The findings, presented at ACR Convergence 2026 in Orlando, were among three studies highlighting significant unmet treatment needs in paediatric rheumatology, including limited approved therapies for childhood-onset systemic lupus erythematosus (cSLE).
In a single-centre case series, researchers investigated autologous anti-CD19 CAR T-cell therapy in eight children with severe or refractory autoimmune disease.
The cohort included four patients with cSLE, three with juvenile dermatomyositis (JDM), and one with juvenile systemic sclerosis (jSSc).
All eight received a single infusion of anti-CD19 CAR T cells, after which all immunosuppressive medications were discontinued.
At the latest follow-up, five patients, including three with cSLE and two with JDM, had achieved drug-free remission, while the remaining three continued to improve without treatment.
Patients with cSLE demonstrated normalisation of complement levels and persistent negativity for antinuclear antibodies and anti-double-stranded DNA antibodies.
All three patients with JDM achieved a major clinical response, while the patient with juvenile systemic sclerosis experienced a reduction in modified Rodnan skin score from 28 to nine at 12 months.
Researchers reported reassuring safety findings, with no disease flares recorded during follow-up.
“These results are encouraging for children with autoimmune diseases that do not respond to conventional treatments,” said study investigator Dr Rebecca Nicolai, a paediatric rheumatologist at IRCCS Ospedale Pediatrico Bambino Gesù in Rome.
“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 paediatric autoimmune diseases.”
The findings remain preliminary, with the small patient cohort and limited follow-up preventing firm conclusions about long-term efficacy and safety.
A separate study presented at the meeting reinforced the urgent need for additional treatment options for children with lupus.
Researchers analysed 1256 patients with cSLE enrolled in the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry across more than 70 North American sites between 2017 and March 2025.
They found that 99.7% had received at least one medication that was not specifically approved for childhood-onset lupus.
Belimumab, approved by the US Food and Drug Administration in 2019, remains the only FDA-approved medication specifically for cSLE, despite the condition’s often aggressive disease course.
Dr Ekemini Ogbu, director of Neuroinflammatory Disease Services in Rheumatology at Cincinnati Children’s Hospital Medical Centre, said the findings demonstrated the substantial treatment gaps facing children with lupus.
“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,” Dr Ogbu said.
“There is a continued urgent need for expanded regulatory approval of therapies for cSLE.”
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A third study examined the performance of the 2006 and 2023 antiphospholipid syndrome (APS) classification criteria in children, an area where accurate classification remains challenging.
The multicentre analysis included 168 children diagnosed with APS and 206 with systemic lupus erythematosus or another systemic autoimmune disease.
Researchers found that the older 2006 criteria demonstrated greater sensitivity and better overall classification performance in paediatric APS, while the newer 2023 criteria were more effective at capturing non-thrombotic manifestations.
The findings suggest that neither set of criteria fully addressed the complexities of paediatric APS, highlighting the importance of further research into classification approaches for children.
Together, the three studies underscored the challenges of treating paediatric rheumatic diseases, from limited regulatory approvals and classification difficulties to the need for innovative therapies in severe, treatment-resistant disease.
The studies were presented at ACR Convergence 2026, held this week in Orlando, Florida.
