ACR Meeting Abstracts

ACR Meeting Abstracts

Abstract Number: 089

Anti-CD19 CAR-T Cell Therapy for the Treatment of Severe/Refractory Pediatric Autoimmune Diseases: A Single-Center Case Series

Claudia Bracaglia1, Rebecca Nicolai2, Marco Becilli3, Emiliano Marasco2, Pietro Merli3, Francesca Del Bufalo3, Virginia Messia2, Maria Isabella Petrone2, Maria Giuseppina Cefalo3, Chiara Rosignoli3, Mattia Algeri3, Franco Locatelli4 and Fabrizio De Benedetti5, 1Division of Rheumatology Ospedale Pediatrico Bambino Gesu' IRCCS, Roma, Italy, Rome, Rome, Italy, 2Division of Rheumatology, Ospedale Pediatrico Bambino Gesù IRCCS, Roma, Italy, Rome, Lazio, Italy, 3Department of Hematology/Oncology, Cell and Gene Therapy, Ospedale Pediatrico Bambino Gesù IRCCS, Roma, Italy, Rome, Lazio, Italy, 4Department of Hematology/Oncology, Cell and Gene Therapy, IRCCS Ospedale Pediatrico Bambino Gesù; Catholic University of the Sacred Heart, Roma, Italy, Rome, Lazio, Italy, 5Bambino Gesu Children's Hospital, Rome, Rome, Italy

Meeting: 2026 Pediatric Rheumatology Symposium

Session Information

Date: Friday, March 20, 2026

Title: Abstracts: Technology

Session Time: 4:37PM-4:42PM

Background/Purpose: CD19 CAR T-cell therapy represents an innovative and promising treatment option in patients with severe and/or refractory autoimmune diseases (AD). The experience in children and adolescents is currently limited. We report on the safety and efficacy of anti-CD19 CAR T-cell therapy in 6 patients with severe and refractory AD: 3 with Juvenile Dermatomyositis (JDM), 2 with childhood Systemic Lupus Erythematosus (cSLE) and 1 with juvenile Systemic Sclerosis (jSSc).

Methods: All 6 patients received a single infusion of fresh, autologous anti-CD19 CAR T-cell product (second-generation lentiviral vector provided by Miltenyi Biotec) manufactured on the CliniMACS ProdigyÒ device (1×106 CAR T cells/kg), after lymphodepletion with cyclophosphamide and fludarabine.

Results: The median follow-up after CAR T-cell therapy is 17 months (range 1-27). Two out of 3 JDM patients had a history of severe chronically active disease with extensive calcinosis and ulcerations; the third patient presented interstitial lung disease (ILD) and persistently active muscle and skin disease with ulcerations. Both cSLE patients had ILD (one with pulmonary hypertension and one with diffuse alveolar hemorrhages) and class V nephritis. One patient had also optic neuritis. The patient with jSSc had diffuse skin involvement, vascular manifestations, ILD, gastrointestinal and cardiac involvement. All patients were refractory to multiple immunosuppressive treatment lines and presented significant side effects from chronic glucocorticoid treatment. In vivo CAR T cells rapidly expanded, reaching a peak with a median number of 42.35 cells per microliter (range 12.86-535.02) after a median of 11 days (range 7-28). CD19+ B cells became undetectable in peripheral blood after a mean of 7 days (median 7, range 5-12). B-cell reconstitution started after a mean of 78 days (median 84, range 42-112). All patients with JDM (follow-up of 27, 12, 9 months respectively) achieved and maintained an ACR–EULAR major clinical response using the Total Improvement Score. Both cSLE patients (follow-up of 27 and 22 months) presented resolution of all signs and symptoms: SLEDAI-2K score (22 at baseline in pt 1; 38 in pt 2) normalized at month 3 (SLEDAI-2K=0). All JDM and cSLE patients are off glucocorticoids and immunosuppressant. The jSSc patient presented stable diffusing capacity for carbon monoxide, forced vital capacity and left ventricular ejection fraction at last follow-up (6 weeks after treatment). Four patients (2 JDM and 2 cSLE) presented a grade 1 cytokine release syndrome (G1), not requiring treatment; one patient with JDM presented a mild (G1) immune effector cell–associated neurotoxicity syndrome (ICANS), characterized by confusion and transient EEG abnormalities, resolving spontaneously after 72 hours. Transient anemia (range G2-G3) and neutropenia (range G3-G4) were recorded in all patients, but cytopenia was limited to the first weeks after infusion. No severe infections were observed.

Conclusion: These data provide evidence of the short- and medium-term efficacy and safety of CD19 CAR T-cell therapy in pediatric patients with refractory AD. 
Reference. Nicolai R et al. Arthritis Rheumatol. 2024 Oct;76(10):1560-1565.

Disclosures: C. Bracaglia: GlaxoSmithKlein(GSK), 6, Novartis, 2, Sobi, 2; R. Nicolai: None; M. Becilli: None; E. Marasco: None; P. Merli: None; F. Del Bufalo: None; V. Messia: None; M. Petrone: None; M. Cefalo: None; C. Rosignoli: None; M. Algeri: None; F. Locatelli: Amgen, 1, 6, Bristol-Myers Squibb(BMS), 6, Gilead, 6, Miltenyi Biomedicine, 6, Novartis, 1, 6, Sanofi, 1, Sobi, 6, Vertex, 1; F. De Benedetti: Abbvie, 2, 5, Apollo, 2, 5, Elixiron, 2, 5, Kiniksa, 2, 5, Novartis, 2, 5, Sanofi, 2, 5, Sobi, 2, 5.

To cite this abstract in AMA style:

Bracaglia C, Nicolai R, Becilli M, Marasco E, Merli P, Del Bufalo F, Messia V, Petrone M, Cefalo M, Rosignoli C, Algeri M, Locatelli F, De Benedetti F. Anti-CD19 CAR-T Cell Therapy for the Treatment of Severe/Refractory Pediatric Autoimmune Diseases: A Single-Center Case Series [abstract]. Arthritis Rheumatol. 2026; 78 (suppl 3). https://acrabstracts.org/abstract/anti-cd19-car-t-cell-therapy-for-the-treatment-of-severe-refractory-pediatric-autoimmune-diseases-a-single-center-case-series/. Accessed .

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