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

被引:4
|
作者
Cabrera-Maqueda, Jose Maria [1 ,2 ,3 ]
Sepulveda, Maria [1 ,2 ,3 ]
Garcia, Raquel Ruiz [1 ,2 ,3 ,4 ]
Munoz-Sanchez, Guillermo [4 ]
Martinez-Cibrian, Nuria [5 ,6 ]
Ortiz-Maldonado, Valentin [5 ,6 ]
Lorca-Arce, Daniel [4 ]
Guasp, Mar [1 ,2 ,3 ]
Llufriu, Sara [1 ,2 ,3 ]
Martinez-Hernandez, Eugenia [1 ,2 ,3 ]
Armangue, Thais [1 ,2 ,3 ]
Fonseca, Elianet G. [2 ,3 ]
Alba-Isasi, Maria Teresa [1 ,2 ,3 ]
Delgado, Julio [3 ,9 ]
Dalmau, Josep [5 ,6 ,7 ,8 ]
Juan, Manel [4 ,10 ]
Saiz, Albert [1 ,2 ]
Blanco, Yolanda [1 ,2 ,3 ]
机构
[1] Hosp Clin Barcelona, Neuroimmunol & Multiple Sclerosis Unit, Serv Neurol, Barcelona, Spain
[2] Univ Barcelona, Barcelona, Spain
[3] Fundacio Recerca Clin Barcelona Inst Invest Biomed, Neuroimmunol Program, Barcelona, Spain
[4] Univ Barcelona, Hosp Clin Barcelona, Inst Invest Biomed August Pi Sunyer i, Dept Immunol, Barcelona, Spain
[5] Hosp Clin Barcelona, Dept Hematol, Barcelona, Spain
[6] Inst Invest Biomed August Pi i Sunyer IDIBAPS, Pediat Neuroimmunol Unit, Barcelona, Spain
[7] Univ Barcelona, St Joan Deu SJD Childrens Hosp, Dept Neurol, Barcelona, Spain
[8] Ctr Invest Biomed Red Canc CIBERONC, Barcelona, Spain
[9] Univ Barcelona, Caixa Res Inst, Barcelona, Spain
[10] Joint Platform Immunotherapy St Joan de Deu Hosp C, Barcelona, Spain
来源
关键词
RELEVANCE;
D O I
10.1212/NXI.0000000000200292
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
ObjectivesIn 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.MethodsCD19-directed CAR T-cells (ARI-0001) were produced in-house by lentiviral transduction of autologous fresh leukapheresis and infused after a conventional lymphodepleting regimen.ResultsA 18-year-old man developed 2 episodes of myelitis associated with serum MOG-IgG, which were followed by 6 episodes of left optic neuritis (ON) and sustained the presence of MOG-IgG over 6 years despite multiple immunotherapies. After the sixth episode of ON, accompanied by severe residual visual deficits, CAR T-cell treatment was provided without complications. Follow-up of cell counts showed complete depletion of CD19+ B cells at day +7; reconstituted B cells at day +141 showing a na & iuml;ve 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.DiscussionThis 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 EvidenceThis provides Class IV evidence. It is a single observational study without controls.
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页数:8
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