PILOT phase II trial final results: Lisocabtagene maraleucel for R/R LBCL in patients not intended for HSCT

Results from the final analysis of the phase II PILOT study (NCT03483103) evaluating lisocabtagene maraleucel (liso-cel), an autologous CD19-directed chimeric antigen receptor (CAR) T-cell therapy, for patients with relapsed/refractory (R/R) large B-cell lymphoma (LBCL) not intended for hematopoietic stem cell transplantation (HSCT) (N = 61) were recently published in Blood Advances by Sehgal et al.

Key data: The overall response rate (ORR) (primary end point) was 80%; 54% achieved a complete response (CR). After a median follow-up of 18.2 months, responses after liso-cel were durable and median overall survival (OS) was not reached. Median progression-free survival (PFS) was 9.0 months (95% confidence interval [CI], 4.2 to not reached), and 18-month PFS and OS rates were 43% (95% CI, 30–55) and 59% (95% CI, 45–70), respectively. Grade ≥3 CRS and neurological events occurred in 2% and 5% of patients, respectively, with no Grade 4 or 5 events.

Key learning: No new safety concerns were identified after longer follow-up, supporting second-line use of liso-cel for patients with LBCL not intended for HSCT. Despite the high incidence of high-grade B-cell lymphoma, primary refractory disease, advanced age, and comorbidities, liso-cel demonstrated a high CR rate and durable CRs, establishing it as an effective second-line therapy for this underserved population with historically poor outcomes.