EHA 2017: Updated CTL019 ELIANA Data Show Durable Remission Rates in Children, Young Adults With Relapsed/Refractory B-Cell ALL
Updated results from the ELIANA clinical trial demonstrated CTL019 (tisagenlecleucel) remission rates are maintained at 6 months in relapsed/refractory pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL). These data from this pivotal trial of CTL019 show that 83% (52 of 63; 95% confidence interval [CI] = 71%–91%) of patients achieved complete remission (CR) or CR with incomplete blood count recovery within 3 months of infusion. No minimal residual disease (MRD) was detected among responding patients. Results from this study of CTL019—an investigational chimeric antigen receptor T cell (CAR-T) therapy—were presented at the 2017 European Hematology Association (EHA) Annual Meeting (Abstract S476).
ELIANA is the first pediatric global CAR-T cell therapy registration trial, with study enrollment having occurred across 25 centers in the US, Canada, European Union, Australia, and Japan. The single-arm, open-label, multicenter phase II study included patients aged 3 to 23 years who were primary refractory, refractory to chemotherapy after their first relapse, relapsed after second line therapy, or ineligible for an allogeneic stem-cell transplant (SCT). Patients in the trial received a median of three prior lines of therapy and 59% of patients had a prior SCT.
Further ELIANA Data
The ELIANA study also showed that the relapse-free probability was 75% (95% CI = 57%–87%; median duration of response not reached) at 6 months and 64% (95% CI = 42%–79%) at 12 months among responders. In addition, the probability of survival was 89% (95% CI = 77%–94%) at 6 months and 79% (95% CI = 63%–89%) at 12 months. The median time from infusion to data cutoff was 8.8 months.
“The updated CTL019 ELIANA data illustrating early observed response rates that have held steady over 6 months' time are exciting findings. Durability is an important measure for children and young adults with relapsed or refractory B-cell ALL, and we are truly encouraged by the results of this study,” said lead investigator Stephan Grupp, MD, PhD, the Yetta Deitch Novotny Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania (Penn), and Director of the Cancer Immunotherapy Frontier Program at the Children's Hospital of Philadelphia (CHOP).
Forty-seven percent of patients in ELIANA experienced grade 3 or 4 cytokine release syndrome (CRS), a known complication of the investigational therapy that may occur when the engineered cells become activated in the patient's body. CRS was managed globally using prior site education on implementation of the CRS treatment algorithm. There were no deaths due to refractory CRS and no incidents of cerebral edema were reported. Fifteen percent of patients experienced grade 3 neurologic events, with no grade 4 events seen.
The ELIANA trial enrolled 88 patients. Of the 88, 16 patients discontinued before infusion and the majority (nine patients) did so due to rapid progression of their disease or deterioration in their clinical status. This reflects the acute and progressive nature of this disease. Of the 16 patients who weren't infused, seven were a result of insufficiently formulated CAR-T cell product. Additionally, five infused patients had not reached 3-month follow-up and four patients were pending infusion at the time of data cutoff.
“These positive, updated ELIANA data help us better understand the ability for CTL019 to maintain durable responses in relapsed/refractory ALL,” said Vas Narasimhan, MD, Global Head of Drug Development and Chief Medical Officer, Novartis. “The results, including relapse-free survival findings at 6 and 12 months, reaffirm our confidence in CTL019 to potentially become an effective treatment for pediatric and young adult patients with relapsed/refractory ALL in need of more options.”
The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.
