TRANSFORM long-term follow-up: Liso-cel for 2L R/R LBCL

TRANSFORM (NCT03575351) was a phase III, global, randomized, open-label trial, evaluating lisocabtagene maraleucel (liso-cel) vs standard of care (SoC) for the second-line (2L) treatment of relapsed/refractory (R/R) large B-cell lymphoma (LBCL; N = 184). Upon completion of TRANSFORM, patients who received liso-cel (either assigned to liso-cel or crossing over from SoC) could enroll in a separate long-term follow-up (LTFU) study (NCT03435796), after up to 3 years of post-randomization follow-up (liso-cel arm) or 12 months post-liso-cel infusion (SoC crossover). Results from this LTFU study of the TRANSFORM trial were published in Blood Advances by Kamdar et al. (liso-cel arm, n = 43; SoC crossover, n = 24).

Key data: With a median follow-up of ~4 years, the combined TRANSFORM and LTFU studies showed a long-term survival benefit with liso-cel; of the 92 patients originally randomized to the liso-cel arm, median overall survival (OS) and progression-free survival (PFS) were not reached (NR), while the 48-month OS and PFS rates were 61.5% (95% confidence interval [CI], 51.2–71.7) and 52.2% (95% CI, 41.5–62.8), respectively. Among patients enrolled in the LTFU study, median OS was NR in both the liso-cel arm and SoC crossover arm, with 24-month OS rates of 95.1% (95% CI, 88.4–100.0) and 78.3% (95% CI, 61.4–95.1), respectively. No new safety signals were reported in the LTFU study; any-grade adverse events (AEs) were reported in 5% of patients in the liso-cel arm and 12.5% of patients in the SoC crossover arm. No liso-cel-related deaths occurred, and no second primary malignancies or serious infections related to liso-cel were observed.

Key learning: After ~4 years of combined follow-up, liso-cel demonstrated durable long-term OS and PFS benefit, and a consistent safety profile, supporting its consideration as a 2L treatment option for eligible patients with R/R LBCL.