Breese Erin H, Krupski Christa, Nelson Adam S, Perentesis John P, Phillips Christine L
Division of Oncology.
Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH.
J Pediatr Hematol Oncol. 2021 May 1;43(4):152-154. doi: 10.1097/MPH.0000000000001857.
Infants with KMT2A-rearranged acute lymphoblastic leukemia (ALL) have historically poor outcomes despite maximal intensification of chemotherapy. Chimeric antigen receptor (CAR) T-cell therapy has revolutionized our approach to pediatric patients with relapsed/refractory ALL. Unfortunately, infants were excluded from early CAR T-cell trials due to concerns regarding the feasibility of T-cell collection and expansion. Here, we report the use of tisagenlecleucel in an infant with chemotherapy-refractory KMT2A-rearranged ALL. While CAR T-cell therapy was not curative for this patient, collection and expansion of T-cells proved feasible despite prior chemotherapy, he achieved minimal residual disease negative remission with excellent quality of life, and it facilitated a delay in hematopoietic stem cell transplantation.
尽管强化化疗已达到最大程度，但患有KMT2A重排急性淋巴细胞白血病（ALL）的婴儿历来预后较差。嵌合抗原受体（CAR）T细胞疗法彻底改变了我们对复发/难治性ALL儿科患者的治疗方法。不幸的是，由于担心T细胞采集和扩增的可行性，婴儿被排除在早期CAR T细胞试验之外。在此，我们报告了替沙格韦单抗在一名患有化疗难治性KMT2A重排ALL的婴儿中的应用。虽然CAR T细胞疗法未能治愈该患者，但尽管之前接受过化疗，T细胞的采集和扩增证明是可行的，他实现了微小残留病阴性缓解，生活质量良好，并且推迟了造血干细胞移植。
