Methods
Pts from TRANSCEND CLL 004 and a matched RW cohort were analyzed. Eligible pts were ≥ 18 y with CLL/SLL, had ≥ 2 prior lines of therapy (pLoT), including a BTKi and venetoclax, and started subsequent tx for CLL/SLL. SOC pts were selected from de-identified datasets (Flatiron [1993–2023], COTA [2000–2023], and ConcertAI [1997–2022]). TRANSCEND CLL 004 eligibility criteria were applied as applicable. Liso-cel pts were eligible trial participants treated with liso-cel and efficacy-evaluable. Endpoints were ORR, PFS, and OS. Inverse probability of tx weighting (IPTW) and regression model were used to balance pt characteristics between cohorts, including age, sex, race, time from initial diagnosis, Rai stage, bulky disease, ECOG PS, high-risk cytogenetics, pLOT, prior chemoimmunotherapy and phosphatidylinositol 3-kinase inhibitor (PI3Ki), and refractoriness to BTKi and venetoclax.
Results
Analysis included 278 pts (SOC, n = 212; liso-cel, n = 66). SOC regimens included chemotherapy, immunotherapy (excluding CAR T cell therapy), BTKi, venetoclax, PI3Ki, and combinations. Median follow-up was 17.2 mo for SOC and 35.4 mo for liso-cel. Most pt characteristics were balanced after IPTW (Table) with imbalance adjusted by regression. After adjustment, ORR (95% CI) was 19.2% (14.1–26.1) for SOC vs 52.5% (34.8–79.2) for liso-cel. Median (95% CI) PFS was 4.4 mo (3.2–5.5) for SOC vs 12.0 mo (10.8–13.2) for liso-cel (HR, 0.40; 95% CI, 0.24–0.68). PFS probabilities at 24 and 36 mo were 11.5% and 5.1% for SOC vs 46.3% and 30.3% for liso-cel. Median (95% CI) OS was 14.8 mo (9.4–20.1) for SOC vs 33.6 mo (31.7–35.5) for liso-cel (HR, 0.47; 95% CI, 0.28–0.79). OS probabilities at 24 and 36 mo were 35.1% and 29.7% for SOC vs 73.4% and 42.6% for liso-cel.
Conclusions
Liso-cel was associated with significantly improved response, delayed progression, and prolonged survival vs SOC in pts with R/R CLL/SLL after ≥ 2 pLOTs, including a BTKi and venetoclax. Clinical trial information: NCT03331198.
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