Müller, 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).
Valenzuela-Almada, M. O. et al. Epidemiology of childhood-onset systemic lupus erythematosus: a population-based study. Arthritis Care Res. 74, 728–732 (2022).
Charras, A., Smith, E. & Hedrich, C. M. Systemic lupus erythematosus in children and young people. Curr. Rheumatol. Rep. 23, 20 (2021).
Ambrose, N. et al. Differences in disease phenotype and severity in SLE across age groups. Lupus 25, 1542–1550 (2016).
Massias, J. S. et al. Clinical and laboratory characteristics in juvenile-onset systemic lupus erythematosus across age groups. Lupus 29, 474–481 (2020).
Md Yusof, M. Y. et al. Management and treatment of children, young people and adults with systemic lupus erythematosus: British Society for Rheumatology guideline scope. Rheumatol. Adv. Pract. 7, rkad093 (2023).
Mendez, E. P. et al. US incidence of juvenile dermatomyositis, 1995–1998: results from the National Institute of Arthritis and Musculoskeletal and Skin Diseases Registry. Arthritis Rheum. 49, 300–305 (2003).
Lu, X., Peng, Q. & Wang, G. Anti-MDA5 antibody-positive dermatomyositis: pathogenesis and clinical progress. Nat. Rev. Rheumatol. 20, 48–62 (2024).
Akgün, G. et al. Cardiac evaluation of patients with juvenile dermatomyositis. Pediatr. Res. 97, 333–340 (2025).
Volkmann, E. R., Andréasson, K. & Smith, V. Systemic sclerosis. Lancet 401, 304–318 (2023).
Foeldvari, I. et al. Best clinical practice in the treatment of juvenile systemic sclerosis: expert panel guidance—the result of the International Hamburg Consensus Meeting December 2022. Expert Rev. Clin. Immunol. 20, 387–404 (2024).
Vasquez-Canizares, N. et al. Developing consensus outcome measures in juvenile systemic sclerosis: a global survey of pediatric rheumatologists and literature review. Pediatr. Rheumatol. Online J. 23, 46 (2025).
Kim, H. Juvenile dermatomyositis: updates in pathogenesis and biomarkers, current treatment, and emerging targeted therapies. Paediatr. Drugs 27, 57–72 (2025).
Northcott, M. et al. Type 1 interferon status in systemic lupus erythematosus: a longitudinal analysis. Lupus Sci. Med. 9, e000625 (2022).
Arbuckle, M. R. et al. Development of autoantibodies before the clinical onset of systemic lupus erythematosus. N. Engl. J. Med. 349, 1526–1533 (2003).
Allenbach, Y. et al. Different phenotypes in dermatomyositis associated with anti-MDA5 antibody: study of 121 cases. Neurology 95, e70–e78 (2020).
Kilinc, O. C. & Ugurlu, S. Clinical features of dermatomyositis patients with anti-TIF1 antibodies: a case based comprehensive review. Autoimmun. Rev. 22, 103464 (2023).
Tansley, S. L. et al. Calcinosis in juvenile dermatomyositis is influenced by both anti-NXP2 autoantibody status and age at disease onset. Rheumatology 53, 2204–2208 (2014).
Hoekstra, E. M. et al. The prognostic power of anti-topoisomerase I and anti-centromere antibodies in systemic sclerosis—a systematic review of the literature. Semin. Arthritis Rheum. 72, 152667 (2025).
Krustev, E., Clarke, A. E. & Barber, M. R. W. B cell depletion and inhibition in systemic lupus erythematosus. Expert Rev. Clin. Immunol. 19, 55–70 (2023).
Merrill, J. T. et al. Efficacy and safety of rituximab in moderately-to-severely active systemic lupus erythematosus: the randomized, double-blind, phase II/III systemic lupus erythematosus evaluation of rituximab trial. Arthritis Rheum. 62, 222–233 (2010).
Brunner, H. I. et al. Safety and efficacy of intravenous belimumab in children with systemic lupus erythematosus: results from a randomised, placebo-controlled trial. Ann. Rheum. Dis. 79, 1340–1348 (2020).
Evans, L. S. et al. Povetacicept, an enhanced dual APRIL/BAFF antagonist that modulates B lymphocytes and pathogenic autoantibodies for the treatment of lupus and other B cell-related autoimmune diseases. Arthritis Rheumatol. 75, 1187–1202 (2023).
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).
Ebata, S. et al. Safety and efficacy of rituximab in systemic sclerosis (DESIRES): open-label extension of a double-blind, investigators-initiated, randomised, placebo-controlled trial. Lancet Rheumatol. 4, e546–e555 (2022).
Gordon, J. K. et al. Belimumab for the treatment of early diffuse systemic sclerosis: results of a randomized, double-blind, placebo-controlled, pilot trial. Arthritis Rheumatol. 70, 308–316 (2018).
Ramwadhdoebe, T. H. et al. Effect of rituximab treatment on T and B cell subsets in lymph node biopsies of patients with rheumatoid arthritis. Rheumatology 58, 1075–1085 (2019).
Leandro, M. J. B-cell subpopulations in humans and their differential susceptibility to depletion with anti-CD20 monoclonal antibodies. Arthritis Res. Ther. 15, S3 (2013).
Rider, L. G. et al. 2016 ACR-EULAR adult dermatomyositis and polymyositis and juvenile dermatomyositis response criteria-methodological aspects. Rheumatology 56, 1884–1893 (2017).
Rider, L. G. et al. Measures of adult and juvenile dermatomyositis, polymyositis, and inclusion body myositis: Physician and Patient/Parent Global Activity, Manual Muscle Testing (MMT), Health Assessment Questionnaire (HAQ)/Childhood Health Assessment Questionnaire (C-HAQ), Childhood Myositis Assessment Scale (CMAS), Myositis Disease Activity Assessment Tool (MDAAT), Disease Activity Score (DAS), Short Form 36 (SF-36), Child Health Questionnaire (CHQ), physician global damage, Myositis Damage Index (MDI), Quantitative Muscle Testing (QMT), Myositis Functional Index-2 (FI-2), Myositis Activities Profile (MAP), Inclusion Body Myositis Functional Rating Scale (IBMFRS), Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI), Cutaneous Assessment Tool (CAT), Dermatomyositis Skin Severity Index (DSSI), Skindex, and Dermatology Life Quality Index (DLQI). Arthritis Care Res. 63, S118–S157 (2011).
De Benedetti, F., Diomedi Camassei, F. & Locatelli, F. CAR T-cell therapy in autoimmune disease. N. Engl. J. Med. 390, 1629 (2024).
Nicolai, R. et al. Autologous CD19-targeting CAR T cells in a patient with refractory juvenile dermatomyositis. Arthritis Rheumatol. 76, 1560–1565 (2024).
Krickau, T. et al. CAR T-cell therapy rescues adolescent with rapidly progressive lupus nephritis from haemodialysis. Lancet 403, 1627–1630 (2024).
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).
París-Muñoz, A. et al. CD19 CAR-T cell therapy in a pediatric patient with MDA5+ dermatomyositis and rapidly progressive interstitial lung disease. Med. 6, 100676 (2025).
Hanif, M. et al. Contributors to organ damage in childhood lupus: corticosteroid use and disease activity. Rheumatology 64, 3028–3038 (2025).
Del Bufalo, F. et al. Point-of-care fresh CAR T cells for pediatric or young adult BCP-ALL that is relapsed/refractory or in very-high-risk first relapse. Blood Adv. 9, 5091–5102 (2025).
Garcia-Prieto, C. A. et al. Epigenetic profiling and response to CD19 chimeric antigen receptor T-cell therapy in B-cell malignancies. J. Natl Cancer Inst. 114, 436–445 (2022).
Talleur, A. C. et al. Preferential expansion of CD8+ CD19-CAR T cells postinfusion and the role of disease burden on outcome in pediatric B-ALL. Blood Adv. 6, 5737–5749 (2022).
Guzzo, I. et al. Anti-CD19 chimeric antigen receptor T-cell therapy in a highly sensitized patient with focal and segmental glomerulosclerosis. Pediatr. Nephrol. 40, 3513–3519 (2025).
Epperly, R. & Shah, N. N. Long-term follow-up of CD19-CAR T-cell therapy in children and young adults with B-ALL. Hematology Am. Soc. Hematol. Educ. Program 2023, 77–83 (2023).
Elsallab, M. et al. Second primary malignancies after commercial CAR T-cell therapy: analysis of the FDA Adverse Events Reporting System. Blood 143, 2099–2105 (2024).
Lamble, A. J. et al. Risk of T-cell malignancy after CAR T-cell therapy in children, adolescents, and young adults. Blood Adv. 8, 3544–3548 (2024).
Youssef, E., Weddle, K., Zimmerman, L. & Palmer, D. Pharmacovigilance in cell and gene therapy: evolving challenges in risk management and long-term follow-up. Drug Saf. 49, 27–53 (2026).
Hayden, P. J. et al. Management of adults and children receiving CAR T-cell therapy: 2021 best practice recommendations of the European Society for Blood and Marrow Transplantation (EBMT) and the Joint Accreditation Committee of ISCT and EBMT (JACIE) and the European Haematology Association (EHA). Ann. Oncol. 33, 259–275 (2022).
Müller, F. et al. CD19-targeted CAR T cells in refractory antisynthetase syndrome. Lancet 401, 815–818 (2023).
Tur, C. et al. CD19-CAR T-cell therapy induces deep tissue depletion of B cells. Ann. Rheum. Dis. 84, 106–114 (2025).
Mackensen, A. et al. Anti-CD19 CAR T cell therapy for refractory systemic lupus erythematosus. Nat. Med. 28, 2124–2132 (2022).
Merkt, W. et al. Persisting CD19.CAR-T cells in combination with nintedanib: clinical response in a patient with systemic sclerosis-associated pulmonary fibrosis after 2 years. Lancet Respir. Med. 13, 651–654 (2025).
Shimizu, M., Nakaseko, H., Muro, Y. & Iwata, N. Effect of abatacept added to mycophenolate mofetil for refractory calcinosis in juvenile dermatomyositis. Mod. Rheumatol. Case Rep. 9, rxaf004 (2025).
Aringer, M. et al. European League Against Rheumatism/American College of Rheumatology classification criteria for systemic lupus erythematosus. Ann. Rheum. Dis. 78, 1151–1159 (2019).
Lundberg, I. E. et al. European League Against Rheumatism/American College of Rheumatology classification criteria for adult and juvenile idiopathic inflammatory myopathies and their major subgroups. Ann. Rheum. Dis. 76, 1955–1964 (2017).
Van den Hoogen, F. et al. Classification criteria for systemic sclerosis: an American College of Rheumatology/European League Against Rheumatism collaborative initiative. Ann. Rheum. Dis. 72, 1747–1755 (2013).
Terrault, N. A. et al. Update on prevention, diagnosis, and treatment of chronic hepatitis B: AASLD 2018 hepatitis B guidance. Hepatology 67, 1560–1599 (2018).
Bhattacharya, D., Aronsohn, A., Price, J. & Lo Re, V. Hepatitis C guidance 2023 update: American Association for the Study of Liver Diseases– Infectious Diseases Society of America recommendations for testing, managing, and treating hepatitis C virus infection. Clin. Infect. Dis. https://doi.org/10.1093/cid/ciad319 (2023).
European Union. Regulation (EC) No 1394/2007 of the European Parliament and of the Council of 13 November 2007 on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004. European Union data.europa.eu/eli/reg/2007/1394/oj (2019).
Del Bufalo, F., Quintarelli, C. & Locatelli, F. Allogeneic CAR T cells: a new player in the field and the peculiar opportunities of the Hospital Exemption path. Mol. Ther. 33, 2326–2327 (2025).
Ambrosone, A. & Cometa, M. F. Hospital Exemption in Italy: technical-scientific and regulatory aspects in comparison with Europe. Cytotherapy 27, 1251–1261 (2025).
Maschan, M. et al. Multiple site place-of-care manufactured anti-CD19 CAR-T cells induce high remission rates in B-cell malignancy patients. Nat. Commun. 12, 7200 (2021).
Lee, D. W. et al. ASTCT consensus grading for cytokine release syndrome and neurologic toxicity associated with immune effector cells. Biol. Blood Marrow Transplant. 25, 625–638 (2019).
Hines, M. R. et al. Immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome. Transplant. Cell. Ther. 29, 438 (2023).
Rejeski, K. et al. Immune effector cell-associated hematotoxicity: EHA/EBMT consensus grading and best practice recommendations. Blood 142, 865–877 (2023).
Gladman, D. D., Ibañez, D. & Urowitz, M. B. Systemic lupus erythematosus disease activity index 2000. J. Rheumatol. 29, 288–291 (2002).
Khanna, D. et al. Standardization of the modified Rodnan skin score for use in clinical trials of systemic sclerosis. J. Scleroderma Relat. Disord. 2, 11–18 (2017).
La Torre, F. et al. A preliminary disease severity score for juvenile systemic sclerosis. Arthritis Rheum. 64, 4143–4150 (2012).
Carsetti, R. et al. Comprehensive phenotyping of human peripheral blood B lymphocytes in healthy conditions. Cytometry A 101, 131–139 (2022).
Carsetti, R. et al. Different innate and adaptive immune responses to SARS-CoV-2 infection of asymptomatic, mild, and severe cases. Front. Immunol. 11, 610300 (2020).
Bagnara, D. et al. A reassessment of IgM memory subsets in humans. J. Immunol. 195, 3716–3724 (2015).
