Three-Year Update of Tisagenlecleucel in Pediatric and Young Adult Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia in the ELIANA Trial.
Level 4 - case-series / case-control
Single-arm phase II clinical trial without a concurrent control group
PubMed 36399695 · doi:10.1200/JCO.22.00642
What was done
This was an updated efficacy, safety, and patient-reported quality-of-life analysis of the global phase II ELIANA trial. Investigators evaluated 79 pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (R/R B-ALL) treated with a single infusion of tisagenlecleucel, with a median follow-up duration of 38.8 months.
What was found
The overall remission rate was 82%. Median event-free survival (EFS) was 24 months, while median overall survival (OS) was not reached. At 3 years, EFS was 44% (95% CI, 31 to 57) and OS was 63% (95% CI, 51 to 73). Estimated 3-year relapse-free survival was 52% (95% CI, 37 to 66) with censoring for subsequent therapy and 48% (95% CI, 34 to 60) without censoring. Grade 3/4 adverse events occurred in 29% of patients more than 1 year post-infusion, with no increase in late grade 3/4 infection rates. Patients experienced sustained improvements in reported quality of life up to 36 months.
Why it matters
The 3-year data demonstrate durable remissions, persistent survival plateaus, and manageable long-term safety in heavily pretreated young patients with relapsed/refractory B-ALL who otherwise have limited therapeutic options.
Limits
The study is an open-label, single-arm trial without a direct randomized comparator. The sample size is modest (n = 79), and specific breakdown of post-infusion subsequent therapies or long-term B-cell aplasia metrics was not fully detailed in the abstract.