The Evolving Role of CAR T Cell Therapy in Hematological Malignancies

A long-term follow-up to the DREPAGREFFE-1 trial suggest that children with sickle cell anemia may benefit long-term on risk of cerebral injury, cognitive functions, and quality of life over standard care transfusions.

Among those who had undetectable minimal residual disease, autologous hematopoietic cell transplantation showed signs of benefit only for those who remained MRD-positive following induction therapy.

Arlocabtagene autoleucel shows promise as a potential first in class GPRC5D-targeted CAR T-cell therapy for heavily pretreated multiple myeloma.

The CAR-T, marketed as Kymriah, showed a 4-year overall survival rate of 79.3% and a median progression-free survival of 53.3 months.

With regard to safety, there were no dose-limiting toxicities, no cases of GvHD, and no cases of TAK-007-related ICANS.

Allo-HSCT showed good 2-year survival data, with matched sibling donors showing superior outcomes to alternative donors.