Table 4.
Comparison of efficacy and toxicity of patients treated with axi-cel outside clinical trials (‘real-world experience’) with ZUMA-1.
| Characteristics | ZUMA-1 (Locke et al. 2018) |
Nastoupil et al.88
(ASH 2018) |
Jacobson et al.89
(ASH 2018) |
|---|---|---|---|
| Patients enrolled (infused), n | 111 (101) | 295 (274) | NR (104) |
| Median age (range), years | 58 (23–76) | 60 (21–83) | 63.8 (21–80) |
| Median follow up | 27.1 months | 3.9 months | 5.6 months |
| Double-hit lymphoma | NR | 23% | 24% |
| ⩾ 3 lines of therapy | 69% | 75% | NR |
| Primary refractoriness | 26% | 35% | NR |
| Refractory to last therapy | 77% | 42% | 91% |
| Prior autologous HCT | 21% | 33% | 27% |
| Bridging chemotherapy | 0 | 55% | 40% |
| Efficacy | |||
| Best ORR (CR) | 82% (58%) | 81% (57%) | 71% (44%) |
| Median PFS | 5.9 months | 6.18 months | 5.6 months |
| 6-month OS | 78% | 72% | NR |
| Toxicity | |||
| CRS all grades (3–4) | 93% (13%) | 92% (7%) | 94% (16%) |
| Neurotoxicity all grades (3–4) | 65% (31%) | 69% (33%) | 76% (39%) |
| Tocilizumab use | 45% | 63% | 67% |
| Steroids use | 29% | 55% | 64% |
| Grade 5 AEs | 4% | 3%1 | 7%2 |
1
A total of 7 nonrelapse mortalities due to: infection (n = 5); hemophagocytic lymphohistiocytosis (n = 1); cerebral edema (n = 1).
2
A total of 7 nonrelapse mortalities due to: CRS (n = 2); neurotoxicity (n = 1); infection (n = 2); cardiovascular (n = 2).
AE, adverse event; ASH 2018, 60th Annual Meeting of the American Society of Hematology; axi-cel, axicabtagene ciloleucel; CR, complete response; CRS, cytokine-releasing syndrome; HCT, hematopoietic cell transplantation; NR, not reported; ORR, overall response rate; OS, overall survival; PFS, progression-free survival.