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.

Regarding these efficacy outcomes, a key study finding was that the results for patients treated with tisagenlecleucel in a real-world setting or a pivotal clinical trial were similar, although “long-term results of time-to-event endpoints need more time to mature,” commented the study authors.

Of note, compared with those patients with ALL enrolled in the pivotal clinical trial, the baseline characteristics of patients with ALL treated in a real world setting included children younger than 3 years, a lower frequency of patients who had previously undergone allogeneic HCT, and more patients with primary refractory disease. A similar comparison for patients with NHL revealed that, in the real world setting, the median patient age was 9 years older and fewer patients had previously undergone autologous HCT.

“The differences in the real-world patient population treated in the registry compared with the patient selection in the pivotal trials highlight the need for more generalizable real-world data in addition to those from clinical trials,” the study authors stated.

Furthermore, the frequency of grade 3 or higher cytokine release syndrome (CRS) reported in the pivotal clinical trial of tisagenlecleucel in ALL was 48.1% compared with only 16.1% for those treated in a real-world setting. Similarly, for NHL, these rates were 22.6% and 4.5%, respectively.

In conclusion, the study investigators commented, “As accrual continues up to 2500 patients followed for 15 years, this [postmarketing requirement] study is well positioned to continue to yield important insights into the use of tisagenlecleucel.”

Disclosures: Some authors have declared affiliations with or received funding from the pharmaceutical industry. Please refer to the original article for a full list of disclosures.


  1. Pasquini MC, Hu Z-H, Curran K, et al. Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma. Blood Adv. 2020;4:5414-5424. doi: 10.1182/bloodadvances.2020003092
  2. Kymriah [package insert]. East Hanover, NJ: Novartis Pharmaceutical Corp; 2017.

This article originally appeared on Oncology Nurse Advisor