Tisagenlecleucel in adult relapsed or refractory follicular lymphoma: the phase 2 ELARA trial.
Level 4 - case-series / case-control
Single-arm phase 2 trial without a concurrent control group
PubMed 34921238 · doi:10.1038/s41591-021-01622-0
What was done
In the single-arm, multinational, phase 2 ELARA trial (NCT03568461), adults with relapsed or refractory follicular lymphoma after two or more lines of therapy or after autologous stem cell transplant received a single infusion of tisagenlecleucel (autologous anti-CD19 CAR-T cell therapy). The primary endpoint was complete response rate (CRR). Secondary endpoints included overall response rate (ORR), duration of response, progression-free survival, overall survival, pharmacokinetics, and safety.
What was found
Of 98 enrolled patients, 97 received tisagenlecleucel with a median follow-up of 16.59 months (IQR 13.8–20.21). In the efficacy analysis (n = 94), the CRR was 69.1% (95% CI 58.8–78.3%) and the ORR was 86.2% (95% CI 77.5–92.4%). Within 8 weeks post-infusion (safety set, n = 97), cytokine release syndrome occurred in 48.5% of patients (0% grade ≥3), neurological events in 37.1% (3% grade ≥3), and immune effector cell-associated neurotoxicity syndrome (ICANS) in 4.1% (1% grade ≥3). There were zero treatment-related deaths.
Why it matters
This trial establishes tisagenlecleucel as a high-efficacy, low-high-grade-toxicity cellular therapy option for heavily pretreated relapsed or refractory follicular lymphoma patients.
Limits
The study is a single-arm trial without a direct comparator or control group. Median follow-up (16.59 months) remains modest for an indolent malignancy, and specific numerical outcomes for duration of response, progression-free survival, and overall survival were not reported in the abstract.
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