CD19-Directed CAR T-Cells in a Patient With Refractory MOGAD

作者：José María Cabrera‐Maqueda, María Sepúlveda, Raquel Ruiz‐García, Guillermo Muñoz‐Sánchez, Núria Martínez‐Cibrián, Valentín Ortiz‐Maldonado, Daniel Lorca‐Arce, Mar Guasp, Sara Llufriú, Eugenia Martínez‐Hernández, Thaís Armangué, Elianet Fonseca, María Teresa Alba-Isasi, Julio García Delgado, Josep Dalmau, Manel Juan, Albert Saiz, Yolanda Blanco · 发表于：Neurology Neuroimmunology & Neuroinflammation · 年份：2024 · DOI：10.1212/nxi.0000000000200292 · 被引用次数：37 · 研究领域：CAR-T cell therapy research、Cutaneous lymphoproliferative disorders research、Lymphoma Diagnosis and Treatment

OBJECTIVES: In MOG antibody-associated disease (MOGAD), relapse prevention and the treatment approach to refractory symptoms are unknown. We report a patient with refractory MOGAD treated with CD19-directed CAR T-cells. METHODS: CD19-directed CAR T-cells (ARI-0001) were produced in-house by lentiviral transduction of autologous fresh leukapheresis and infused after a conventional lymphodepleting regimen. RESULTS: B cells at day +7; reconstituted B cells at day +141 showing a naïve B-cell phenotype, and low or absent memory B cells and plasmablasts for 1 year. MOG-IgG titers have remained undetectable since CAR T-cell infusion. The patient had an early episode of left ON at day +29, when MOG-IgG was already negative, and since then he has remained free of relapses without immunotherapy for 1 year. DISCUSSION: This clinical case shows that CD19-directed CAR T-cell therapy is well-tolerated and is a potential treatment for patients with refractory MOGAD. CLASSIFICATION OF EVIDENCE: This provides Class IV evidence. It is a single observational study without controls.