Chimeric antigen receptor T-cell therapy for marrow and extramedullary relapse of infant acute lymphoblastic leukemia

Chimeric antigen receptor (CAR) T-cells, engineered autologous T-cells that target antigens found in leukemia, have shown durable remissions in relapsed acute lymphoblastic leukemia (ALL). Infant ALL withKMT2Arearrangements (KMT2Ar) is a rare, aggressive form of leukemia associated with extramedullary disease both at diagnosis and at relapse, and overall outcomes for these patients are dismal. Here we report the successful use of tisagenlecleucel, a CAR T-cell product approved for relapsed/refractory ALL, in a patient withKMT2Ar infant ALL who was treated for combined marrow and extramedullary (renal) relapse.

Automated summary

CAR T-cell therapy has shown successful application in patients with KMT2Ar infant ALL, offering potential efficacy for patients with extramedullary disease.