Short-term follow-up of the efficacy and safety of the chimeric antigen receptor T-cell (CAR-T) therapy tisagenlecleucel revealed clinical outcomes that were similar to those observed in pivotal clinical trials evaluating the therapy. This analysis included patients with acute lymphoblastic leukemia (ALL) and non-Hodgkin lymphoma (NHL) treated with tisangelecleucel in a real-world setting. These results were published in Blood Advances.1

Tisagenlecleucel became commercially available in the United States in 2017 for the treatment of patients aged 25 years or younger with relapsed/refractory B-cell precursor ALL and adult patients with relapsed/refractory large B-cell lymphoma.2

This analysis of patients included in the database of the non-hematopoietic cell transplantation cellular therapy (CT) registry from the Center for International Blood and Marrow Transplant Research (CIBMTR) evaluated the clinical outcomes of patients who received tisagenlecleucel for an approved indication in either the United States or Canada.


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Of the 410 patients who had been infused with tisagenlecleucel outside the setting of a clinical trial between August 30, 2017, and January 23, 2020, for whom 3-month follow-up data were prospectively collected following their inclusion in the CT, 255 and 155 patients had a diagnosis of ALL and NHL, respectively. Data related to disease outcome were available for only 401 of these patients; hence, efficacy analyses were conducted using this latter patient subgroup.

“This report represents the largest published series of patients with ALL and NHL treated with tisagenlecleucel,” the study investigators noted.

The median follow-up for young patients with ALL was 13.4 months, and the initial complete remission (CR) rate was 85%. In addition, 12-month rates of duration of response (DOR), event-free survival (EFS), and overall survival (OS) were 60.9%, 52.4%, and 77.2%, respectively.

For adult patients with NHL, median follow-up was 11.9 months. The best overall response rate was 61.8%, with an initial CR of 39.5%, and 6-month rates of DOR, progression-free survival, and OS of 55.3%, 38.7%, and 70.7%, respectively.