Pasquini, M. C. et al. Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma. Blood Adv. 4, 5414–5424 (2020).
Article CAS PubMed PubMed Central Google Scholar
Boardman, A. P. & Salles, G. CAR T-cell therapy in large B cell lymphoma. Hematol. Oncol. 41, 112–118 (2023).
Article PubMed PubMed Central Google Scholar
Gardner, R. A. et al. Intent-to-treat leukemia remission by CD19 CAR T cells of defined formulation and dose in children and young adults. Blood 129, 3322–3331 (2017).
Article CAS PubMed PubMed Central Google Scholar
Maude, S. L. et al. Tisagenlecleucel in children and young adults with B-cell lymphoblastic leukemia. N. Engl. J. Med. 378, 439–448 (2018).
Article CAS PubMed PubMed Central Google Scholar
Haghikia, A. et al. Anti-CD19 CAR T cells for refractory myasthenia gravis. Lancet Neurol. 22, 1104–1105 (2023).
Article CAS PubMed Google Scholar
Auth, J. et al. CD19-targeting CAR T-cell therapy in patients with diffuse systemic sclerosis: a case series. Lancet Rheumatol. 7, e83–e93 (2024).
Qin, C. et al. Single-cell analysis of refractory anti-SRP necrotizing myopathy treated with anti-BCMA CAR-T cell therapy. Proc. Natl Acad. Sci. USA 121, e2315990121 (2024).
Article CAS PubMed PubMed Central Google Scholar
Lodka, D. et al. CD19-targeting CAR T cells protect from ANCA-induced acute kidney injury. Ann. Rheum. Dis. 83, 499–507 (2024).
Article CAS PubMed Google Scholar
Lee, J. et al. Antigen-specific B cell depletion for precision therapy of mucosal pemphigus vulgaris. J. Clin. Invest. 130, 6317–6324 (2020).
Article CAS PubMed PubMed Central Google Scholar
Sadun, R. E. & Foster, M. H. Deja vu but new: using T cells to deplete B cells to treat lupus. Am. J. Kidney Dis. 74, 708–710 (2019).
Kansal, R. et al. Sustained B cell depletion by CD19-targeted CAR T cells is a highly effective treatment for murine lupus. Sci. Transl. Med. 11, eaav1648 (2019).
Article PubMed PubMed Central Google Scholar
Muller, F. et al. CD19 CAR T-cell therapy in autoimmune disease — a case series with follow-up. N. Engl. J. Med. 390, 687–700 (2024).
Wilkinson, M. G. L. & Rosser, E. C. B cells as a therapeutic target in paediatric rheumatic disease. Front. Immunol. 10, 214 (2019).
Article PubMed PubMed Central Google Scholar
Liu, T. et al. Spatial transcriptomics identifies cellular and molecular characteristics of scleroderma skin lesions: pilot study in juvenile scleroderma. Int. J. Mol. Sci. 25, 9182 (2024).
Article CAS PubMed PubMed Central Google Scholar
Torok, K. S. Updates in systemic sclerosis treatment and applicability to pediatric scleroderma. Rheum. Dis. Clin. North. Am. 47, 757–780 (2021).
Bloom, J. L. & Wu, E. Y. Update on antineutrophil cytoplasmic autoantibody vasculitis in children. Curr. Opin. Rheumatol. 36, 336–343 (2024).
Article CAS PubMed Google Scholar
Mahmoud, I. et al. Efficacy and safety of rituximab in the management of pediatric systemic lupus erythematosus: a systematic review. J. Pediatr. 187, 213–219.e2 (2017).
Article CAS PubMed Google Scholar
Balevic, S. J. et al. Extrapolation of adult efficacy data to pediatric systemic lupus erythematosus: evaluating similarities in exposure-response. J. Clin. Pharmacol. 63, 105–118 (2023).
Article CAS PubMed Google Scholar
Sherman, M. A. et al. Treatment escalation patterns to start biologics in refractory moderate juvenile dermatomyositis among members of the Childhood Arthritis and Rheumatology Research Alliance. Pediatr. Rheumatol. Online J. 21, 3 (2023).
Article PubMed PubMed Central Google Scholar
Oddis, C. V. et al. Rituximab in the treatment of refractory adult and juvenile dermatomyositis and adult polymyositis: a randomized, placebo-phase trial. Arthritis Rheum. 65, 314–324 (2013).
Article CAS PubMed PubMed Central Google Scholar
Aggarwal, R. et al. Predictors of clinical improvement in rituximab-treated refractory adult and juvenile dermatomyositis and adult polymyositis. Arthritis Rheumatol. 66, 740–749 (2014).
Article CAS PubMed PubMed Central Google Scholar
Gagne, S. J. et al. Comparing rituximab and cyclophosphamide in induction therapy for childhood-onset anti-neutrophil cytoplasmic antibody-associated vasculitis: an ARChiVe registry cohort study. Arthritis Care Res. 77, 504–512 (2025).
Jamois, C. et al. Rituximab pediatric drug development: pharmacokinetic and pharmacodynamic modeling to inform regulatory approval for rituximab treatment in patients with granulomatosis with polyangiitis or microscopic polyangiitis. Clin. Transl. Sci. 15, 2172–2183 (2022).
Article CAS PubMed PubMed Central Google Scholar
Md Yusof, M. Y. et al. Predicting and managing primary and secondary non-response to rituximab using B-cell biomarkers in systemic lupus erythematosus. Ann. Rheum. Dis. 76, 1829–1836 (2017).
Wobma, H., Chang, J. C. & Prockop, S. E. Releasing our model T — chimeric antigen receptor (CAR) T-cells for autoimmune indications. Curr. Opin. Rheumatol. 37, 128–135 (2025).
Article CAS PubMed Google Scholar
Tur, C. et al. CD19-CAR T-cell therapy induces deep tissue depletion of B cells. Ann. Rheum. Dis. 84, 106–114 (2025).
Article CAS PubMed Google Scholar
Krickau, T. et al. CAR T-cell therapy rescues adolescent with rapidly progressive lupus nephritis from haemodialysis. Lancet 403, 1627–1630 (2024).
Article CAS PubMed Google Scholar
He, X. et al. Treatment of two pediatric patients with refractory systemic lupus erythematosus using CD19-targeted CAR T-cells. Autoimmun. Rev. 24, 103692 (2025).
Article CAS PubMed Google Scholar
Nicolai, R. et al. Autologous CD19-targeting CAR T cells in a patient with refractory juvenile dermatomyositis. Arthritis Rheumatol. 76, 1560–1565 (2024).
Schett, G. et al. Advancements and challenges in CAR T cell therapy in autoimmune diseases. Nat. Rev. Rheumatol. 20, 531–544 (2024).
Chung, J. B., Brudno, J. N., Borie, D. & Kochenderfer, J. N. Chimeric antigen receptor T cell therapy for autoimmune disease. Nat. Rev. Immunol. 24, 830–845 (2024).
Article CAS PubMed PubMed Central Google Scholar
Stojkic, I. et al. CAR T cell therapy for refractory pediatric systemic lupus erythematosus: a new era of hope? Pediatr. Rheumatol. Online J. 22, 72 (2024).
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