DISCUSSION
This case highlights that apheresis for CAR-T cell generation can be done safely and successfully in an infant. Secondly, use of a product with modified CAR-T parameters can result in remission. Despite not meeting the FDA’s percent of CAR positive viable cell specifications, our patient’s manufactured CAR-T product was equally capable of expansion and leukemia targeting with no evidence of greater safety risk.
Lastly, our patient’s ongoing remission status cannot be stressed enough. The availability of tisagenlecleucel provided an opportunity to control very chemotherapy resistant disease prior to HSCT. Pre-transplant remission status following second-line therapy significantly impacts survival following HSCT in patients with r/r KMT2A-r ALL;[3,8] the added benefit of tisagenlecleucel facilitated a deeper remission prior to receiving HSCT, thereby providing an opportunity for maximal long-term survival in an infant who previously had a dismal prognosis. With her being approximately 12-months in remission post-HSCT, she is now beyond the greatest risk period for relapse.[3] Future clinical investigations using CAR-T therapy prior to HSCT for infants with r/r KMT2A-r ALL offers the potential for improved survival in a disease where little progress has been made in treatment outcomes.