Tisagenlecleucel versus historical standard therapies for pediatric relapsed/refractory acute lymphoblastic leukemia.

We compared outcomes from a single-arm study of tisagenlecleucel with standard of care (SOC) regimens in pediatric and young adult patients with relapsed/refractory acute lymphoblastic leukemia (ALL). The analysis included one tisagenlecleucel study, one blinatumomab study, one clofarabine monotherapy study, three studies of clofarabine combination regimens and two studies of other salvage chemotherapy. Matching-adjusted indirect comparison analyses were conducted. After adjusting for baseline characteristics, tisagenlecleucel was associated with significantly prolonged overall survival compared with blinatumomab (hazard ratio [95% CI], 0.32 [0.16-0.64]); clofarabine monotherapy (0.24 [0.13-0.42]); clofarabine combination regimens (0.26 [0.15-0.45]); two salvage therapies (0.15 [0.09-0.25] and 0.27 [0.15-0.49]). The analysis demonstrated tisagenlecleucel was associated with substantially greater survival benefit versus all SOC regimens.