Table 1.
Treatment Disposition and Baseline Characteristics of the Patients.*
| Variable | Patients with DLBCL |
Patients with PMBCL or TFL |
All Patients |
|---|---|---|---|
| Treatment disposition | |||
| No. of patients enrolled | 81 | 30 | 111 |
| Treatment with axi-cel — no. (%) | |||
| Yes | 77 (95) | 24 (80) | 101 (91) |
| No | 4 (5) | 6 (20) | 10 (9) |
| Death before treatment† | 1 (1) | 2 (7) | 3 (3) |
| Adverse events‡ | 3 (4) | 2 (7) | 5 (5) |
| Other§ | 0 | 2 (7) | 2 (2) |
| Characteristics at baseline | |||
| No. of patients | 77 | 24 | 101 |
| Disease type — no. (%) | |||
| DLBCL | 77 (100) | 0 | 77 (76) |
| PMBCL | 0 | 8 (33) | 8 (8) |
| TFL | 0 | 16 (67) | 16 (16) |
| Age | |||
| Median (range) — yr | 58 (25–76) | 57 (23–76) | 58 (23–76) |
| ≥65 yr — no. (%) | 17 (22) | 7 (29) | 24 (24) |
| Male sex — no. (%) | 50 (65) | 18 (75) | 68 (67) |
| ECOG performance-status score of 1 — no. (%) | 49 (64) | 10 (42) | 59 (58) |
| Disease stage — no. (%) | |||
| I or II | 10 (13) | 5 (21) | 15 (15) |
| III or IV | 67 (87) | 19 (79) | 86 (85) |
| International Prognostic Index score — no. (%)¶ | |||
| 0–2 | 40 (52) | 13 (54) | 53 (52) |
| 3 or 4 | 37 (48) | 11 (46) | 48 (48) |
| CD-19 status — no./total no. (%)‖ | |||
| Negative | 7/63 (11) | 1/19 (5) | 8/82 (10) |
| Positive | 56/63 (89) | 18/19 (95) | 74/82 (90) |
| Prior therapies — no. (%) | |||
| ≥Three prior lines of therapy | 49 (64) | 21 (88) | 70 (69) |
| History of primary refractory disease** | 23 (30) | 3 (12) | 26 (26) |
| History of resistance to two consecutive lines | 39 (51) | 15 (62) | 54 (53) |
| Refractory subgroup at study entry — no. (%) | |||
| Primary refractory | 2 (3) | 0 | 2 (2) |
| Refractory to second-line or subsequent therapy | 59 (77) | 19 (79) | 78 (77) |
| Relapse after autologous stem-cell transplantation | 16 (21) | 5 (21) | 21 (21) |
The abbreviation axi-cel denotes axicabtagene ciloleucel, DLBCL diffuse large B-cell lymphoma, ECOG Eastern Cooperative Oncology Group, PMBCL primary mediastinal large B-cell lymphoma, and TFL transformed follicular lymphoma.
Two patients died from disease progression (one after unsuccessful manufacture of the CAR T-cell product) and one from the tumor lysis syndrome.
The adverse events in the four patients who had undergone leukapheresis but had not received conditioning therapy or axi-cel were small intestine obstruction, hypoxia and pleural effusion, spinal column stenosis, and deep-vein thrombosis. The remaining patient received conditioning therapy but had a skin and wound infection that led to ecthyma and sepsis before axi-cel treatment.
The two patients in this category had nonmeasurable disease after leukapheresis.
Scores on the International Prognostic Index include low risk (0 or 1 point), low-intermediate risk (2 points), high-intermediate risk (3 points), and high risk (4 or 5 points).
The CD19 histologic score was assessed in the 82 patients with available samples.
Patients may have had other therapies after primary refractory disease.