CD19 CAR T cells for infants and young children
Menée dans 10 pays européens à partir de données portant sur 35 enfants atteints d'une leucémie lymphoblastique aiguë à cellules B et réfractaire ou récidivante (âge au moment du dépistage de la maladie : moins de 3 ans), cette étude de cohorte rétrospective évalue l'efficacité, du point de vue de la survie globale, de la survie sans événement et de l'aplasie des cellules B, et la toxicité du tisagenlecleucel
With the remarkable ability of CD19-targeted chimeric antigen receptor (CAR) T cellsto overcome chemotherapeutic resistance in B-cell acute lymphoblastic leukaemia, optimallyextending this therapy to improve outcomes in patients with disease that is difficultto treat is imperative. Infants with B-cell acute lymphoblastic leukaemia (aged <1year at diagnosis) are among the most challenging patients to treat, because the leukaemiais frequently refractory to chemotherapy, and intensification with standard chemotherapyhas yielded little improvement in outcomes. With an incidence of 41 cases per million children in the USA, infant acute lymphoblasticleukaemia is rare; however, the disease can be aggressive, and infants often present with hyperleukocytosis,hepatosplenomegaly, extramedullary disease, or CNS involvement, or a combination thereof. Additionally, a large proportion of infant leukaemias show KMT2A gene rearrangements, which often confer chemotherapy resistance. Even with use ofallogeneic haematopoietic stem-cell transplantation (HSCT) in infants with high-riskdisease, the 4-year disease-free survival is only 44%, highlighting the need for novel therapies. At present, there is no consensus on salvagetreatments for patients with relapsed or refractory disease.
The Lancet Haematology , commentaire, 2021