Successful therapy of chimeric antigen receptor T cells for isolated extramedullary acute lymphoblastic leukemia

[摘要] Despite many clinical trials of CD19-targeted CAR T cells for thetreatment of B-cell acute lymphoblastic leukemia (B-ALL) having beenreported, few studies of CD19 CAR T therapy for isolated refractory/relapsed (r/r) extramedullary ALL (EM-ALL) patients have beenpublished. Extramedullary involvement is considered an unfavorableprognostic factor, which often reduces leukemia response to inductionchemotherapy and is usually associated with shorter progression-freesurvival (PFS) and overall survival (OS) [1, 2]. There is still no standardguideline on the treatment of isolated EM-ALL [3]. Considering theabsence of leukemia blasts in the peripheral blood and bone marrowin isolated EM-ALL patients, the questions of whether CAR T cells cansuccessfully expand in these patients and whether these patients canbenefit from CD19 CAR T therapy remain unanswered. In this study,we evaluated the safety and efficacy of CD19 CAR T cells for isolatedr/r EM-ALL patients (ChiCTR2000038532).