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CAR T Cell Data for Pediatric and Young Adult Patients With Relapsed/Refractory ALL

Stephan Grupp, MD, PhD
Published Online:9:41 PM, Tue January 8, 2019


Stephan Grupp, MD, PhD, director of translational research for the Center for Childhood Cancer Research at the Children's Hospital of Philadelphia (CHOP) and medical director of the Stem Cell Laboratory at CHOP, discusses updated findings from the phase II ELIANA trial of tisagenlecleucel (Kymriah), a chimeric antigen receptor (CAR) T-cell therapy, in pediatric and young adult patients with relapsed/refractory acute lymphoblastic leukemia (ALL) with high disease burden.

The primary endpoint of the trial was to put these patients into remission, Grupp says. According to the data, around 82% of patients went into remission. At 12- and 18-month follow-ups, 66% of patients remained in remission.

At a median follow-up of 24 months, about 62% of patients were still in remission. Grupp says these data are interesting because as a specialist in bone marrow transplant, he wants patients to remain in remission rather than go on to transplant. He notes that only a small number of patients have went on to transplant.
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