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.