BackgroundSystemic Lupus Erythematosus (SLE) is a rare and highly heterogeneous autoimmune disease (AD), where standard treatment is based on corticosteroids and conventional or biological immunosuppressive drugs. In severe SLE patients, resistant to 1st and 2nd line therapies, the 10-year mortality remains around 10-15%. Autologous anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapy has recently demonstrated sustained remission in this subset of SLE patients, but real-word treated cases remain low.ObjectiveTo assess Clinical Practices (CPs) and the various challenges in recruiting severe SLE patients eligible for CAR-T cell therapy.MethodsA one year retrospective multicenter study (February 2024–February 2025) was conducted across seven certified AD reference centers. All adult SLE patients fulfilling the 2019 EULAR/ACR criteria were screened for disease activity and severity according to the 2024 EBMT-ISCT expert consensus. Eligibility for CAR-T or other non-cell and gene (CGT) therapy trials and reasons for non-inclusion were analyzed.ResultsAmong 1,844 SLE patients, 54 (2.9%) demonstrated severe disease criteria. Of these, 49 patients were not selected for CAR-T trials, due to either disease remission, lack of specific autoantibody, participation in other trials, physician/patient refusal or exclusion criteria at enrollment and three (1.8%) were ultimately treated.ConclusionsRetrospective analysis of CPs showed that very few severe SLE patients were enrolled for CAR-T trial despite eligibility criteria. Streamlined referral pathways, multidisciplinary coordination, and improved physician education are needed to enhance access to advanced CGT therapies. Keywords : CAR-T cell; SLE; Lupus; Cell and Gene therapy (CGT) Acknowledgements “This work was supported as part of the national plan for rare diseases by the French Ministry of Health, FAI2R”
- Systemic Lupus Erythematosus (SLE) patients eligible for CAR-T cell therapy following the 2024 EBMT and ISCT expert-based recommendations: a one-year clinical practice study in France
- Martin Nivet - Université de BourgogneIngrid Munia - Centre d'Immunologie et des Maladies InfectieusesBenjamin Crichi - Centre d'Immunologie et des Maladies InfectieusesTamim Alsuliman - Centre Hospitalier de Saint-QuentinRoberta Di Blasi - Hôpital Saint-LouisMohamad Sabbah - Hôpital Saint-LouisErwan Le Tallec - InsermSondess Hadj-KhelifaClément Beuvon - Centre Hospitalier Universitaire de PoitiersCatney Charles - Université de Montpellier, Cellules souches, plasticité cellulaire, régénération tissulaire et immunothérapie des maladies inflammatoires - IRMBMarie Jachiet - Assistance Publique – Hôpitaux de ParisLouis Terriou - Centre Hospitalier Universitaire de LilleGrégory PugnetAlexandre Thibault Jacques MariaRobin Dhôte - Hôpital AvicennePedro Henrique Prata - Hôpital DupuytrenZora Marjanovic - Assistance Publique – Hôpitaux de ParisMickey KohCatherine Thieblemont - Hôpital Saint-LouisDominique Farge - Inserm
- Cytotherapy
- 99187703209311
- Cellules souches, plasticité cellulaire, régénération tissulaire et immunothérapie des maladies inflammatoires - IRMB
- English
- Journal article
- hal-05493449