‘Great news’ according to researchers
Hailed as a “true game changer” for pediatric leukemia, Chimeric antigen receptor (CAR) T-cell therapy is now supported by new data showing that this novel therapy can be used even in the smallest of patients ― one-year-old babies.
The results come from a small cohort of infants with relapsed or refractory KMT2A-rearranged infant B-cell acute lymphoblastic leukemia (ALL). Of the 16 patients who were treated, 71.4 percent remain leukemia free at 1 year. Overall survival is 73.3 percent.
The CAR T-cell therapies were as effective for younger patients as has been observed for older children, and there was no increase in toxicities as compared to older patients. Thus, despite theoretical concerns that infants may not do as well or greater safety concerns with infants, “that hasn’t been our experience,” commented lead author Colleen Annesley, MD, an attending physician at Seattle Children’s Hospital and an assistant professor in the Department of Pediatrics at the University of Washington, Seattle. “It’s great news.”
The study was presented here at American Society of Hematology (ASH) 2019. “We know that babies do poorly with leukemia, and those that relapse have a dismal chance of survival,” Annesley told Medscape Medical News.
For infants with relapsed ALL, the reported three-year overall survival rate is about 21%.
“We thought that CAR T cells could salvage some of them, but there were a lot of concerns about using them in this population,” she commented.
Infants present unique challenges in comparison with older patients. Smaller patient size, heavily pretreated disease, and high leukemia burden are characteristic of these patients, and these factors can make apheresis and the manufacture of a T-cell product more difficult.
The treatment is being hailed as “effective and safe.”