ZUMA-5（axi-cel）对比 SCHOLAR-5 外部对照在复发/难治性滤泡性淋巴瘤中的比较有效性

In the pivotal ZUMA-5 trial, axicabtagene ciloleucel (axi-cel; an autologous anti-CD19 chimeric antigen receptor T-cell therapy) demonstrated high rates of durable response in relapsed/refractory (r/r) follicular lymphoma (FL) patients.

Here, outcomes from ZUMA-5 are compared with the international SCHOLAR-5 cohort, which applied key ZUMA-5 trial eligibility criteria simulating randomized controlled trial conditions. SCHOLAR-5 data were extracted from institutions in 5 countries, and from 1 historical clinical trial, for r/r FL patients who initiated a third or higher line of therapy after July 2014.

Patient characteristics were balanced through propensity scoring on prespecified prognostic factors using standardized mortality ratio (SMR) weighting. Time-to-event outcomes were evaluated using weighted Kaplan-Meier analysis.

Overall response rate (ORR) and complete response (CR) rate were compared using weighted odds ratios. The 143 ScHOLAR-5 patients reduced to an effective sample of 85 patients after SMR weighting vs 86 patients in ZUMA-5. Median follow-up time was 25. 4 and 23. 3 months for SCHOLAR-5 and ZUMA-5. Median overall survival (OS) and progression-free survival (PFS) in SCHOLAR-5 were 59. 8 months and 12. 7 months and not reached in ZUMA-5. Hazard ratios for OS and PFS were 0.

42 (95% confidence interval [CI], 0. 21-0. 83) and 0. 30 (95% CI, 0. 18-0. 49). The ORR and CR rate were 49. 9% and 29. 9% in SCHOLAR-5 and 94. 2% and 79. 1% in ZUMA-5, for odds ratios of 16. 2 (95% CI, 5. 6-46. 9) and 8. 9 (95% CI, 4. 3-18. 3). Compared with available therapies, axi-cel demonstrated an improvement in meaningful clinical endpoints, suggesting axi-cel addresses an important unmet need for r/r FL patients. This trial was registered at www. clinicaltrials. gov as #NCT03105336.