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.