Cohort characteristics and tisagenlecleucel details
| . | All (n = 13) . | Responders (n = 10) . | Nonresponders (n = 3) . |
|---|---|---|---|
| Age at CART, median (range), y | 20 (7-24) | 17 (7-23) | 24 (23-24) |
| Diagnosis | |||
| B-LLy | 6 | 5 | 1 |
| BL | 2 | 1 | 1 |
| DLBCL, non-PTLD | 1 | 1 | 0 |
| DLBCL, PTLD | 2 | 2 | 0 |
| PMBCL | 2 | 1 | 1 |
| Stage at diagnosis | |||
| II | 1 | 0 | 1 |
| III | 6 | 5 | 1 |
| IV | 5 | 4 | 1 |
| Unknown | 1 | 1 | 0 |
| Prior lines of therapies, median (range) | 3 (1-8) | 3 (1-8) | 5 (3-6) |
| Prior allogeneic HSCT | 3 | 2 | 1 |
| Disease status at CART | |||
| Primary refractory | 7 | 5 | 2 |
| Second or greater relapse | 6 | 5 | 1 |
| ALC at collection, median (range), x 103/μL | 0.78 (0.30-2.5) | 0.86 (0.45-2.5) | 0.71 (0.30-2.5) |
| CNS involvement at time of CART | 3 | 2 | 1 |
| Cerebrospinal fluid positive | 2 | 1 | 1 |
| Parenchymal disease | 1 | 1 | 0 |
| CART cell dose infused, median (range), ×106 cells per kg | 3.4 (1.2-9.5) | 3.4 (1.2-9.5) | 3.8 (2.1-6.3) |
| CART product type | |||
| Managed access program∗ | 1 | 1 | 0 |
| Release criteria met | 12 | 9 | 3 |
| Lymphodepletion regimen | |||
| Fludarabine/cyclophosphamide | 11 | 10 | 1 |
| Bendamustine | 2 | 0 | 2 |
| CRS | |||
| Any grade | 10 | 8 | 2 |
| Grade 3† | 2 | 2 | 0 |
| Time to CRS, median (range), d | 3 (1-7) | 2.5 (1-4) | 5.5 (4-7) |
| CRS duration, median (range), d | 5.5 (2-13) | 6.5 (2-13) | 2 (2-2) |
| CRS-directed therapy | |||
| Tocilizumab | 3 | 3 | 0 |
| Steroids | 2 | 2 | 0 |
| Neurotoxicity, grade 3‡ | 1 | 1 | 0 |
| Best CART response | |||
| CR | 7 | 7 | 0 |
| PR | 3 | 3 | 0 |
| No response/stable disease | 2 | 0 | 2 |
| Progressive disease | 1 | 0 | 1 |
| Post-CART therapies | |||
| None | 5 | 5 | 0 |
| HSCT | |||
| During CART-mediated remission | 2 | 2§ | 0 |
| Salvage for R/R disease | 3 | 2§ | 1 |
| Pembrolizumab | 2 | 2 | 0 |
| Salvage systemic therapy | 5 | 3 | 2 |
| Investigational CART therapy | 1 | 1 | 0 |
| Status at last follow-up | |||
| Alive, no disease | 8 | 8 | 0 |
| Alive, with disease | 2 | 2 | 0 |
| Deceased | 3 | 0 | 3 |
| Time after CART at last follow-up median (range), mo | 21.2 (0.9-54) | 25.4 (0.9-54) | 9.8 (3.2-19.7) |
| . | All (n = 13) . | Responders (n = 10) . | Nonresponders (n = 3) . |
|---|---|---|---|
| Age at CART, median (range), y | 20 (7-24) | 17 (7-23) | 24 (23-24) |
| Diagnosis | |||
| B-LLy | 6 | 5 | 1 |
| BL | 2 | 1 | 1 |
| DLBCL, non-PTLD | 1 | 1 | 0 |
| DLBCL, PTLD | 2 | 2 | 0 |
| PMBCL | 2 | 1 | 1 |
| Stage at diagnosis | |||
| II | 1 | 0 | 1 |
| III | 6 | 5 | 1 |
| IV | 5 | 4 | 1 |
| Unknown | 1 | 1 | 0 |
| Prior lines of therapies, median (range) | 3 (1-8) | 3 (1-8) | 5 (3-6) |
| Prior allogeneic HSCT | 3 | 2 | 1 |
| Disease status at CART | |||
| Primary refractory | 7 | 5 | 2 |
| Second or greater relapse | 6 | 5 | 1 |
| ALC at collection, median (range), x 103/μL | 0.78 (0.30-2.5) | 0.86 (0.45-2.5) | 0.71 (0.30-2.5) |
| CNS involvement at time of CART | 3 | 2 | 1 |
| Cerebrospinal fluid positive | 2 | 1 | 1 |
| Parenchymal disease | 1 | 1 | 0 |
| CART cell dose infused, median (range), ×106 cells per kg | 3.4 (1.2-9.5) | 3.4 (1.2-9.5) | 3.8 (2.1-6.3) |
| CART product type | |||
| Managed access program∗ | 1 | 1 | 0 |
| Release criteria met | 12 | 9 | 3 |
| Lymphodepletion regimen | |||
| Fludarabine/cyclophosphamide | 11 | 10 | 1 |
| Bendamustine | 2 | 0 | 2 |
| CRS | |||
| Any grade | 10 | 8 | 2 |
| Grade 3† | 2 | 2 | 0 |
| Time to CRS, median (range), d | 3 (1-7) | 2.5 (1-4) | 5.5 (4-7) |
| CRS duration, median (range), d | 5.5 (2-13) | 6.5 (2-13) | 2 (2-2) |
| CRS-directed therapy | |||
| Tocilizumab | 3 | 3 | 0 |
| Steroids | 2 | 2 | 0 |
| Neurotoxicity, grade 3‡ | 1 | 1 | 0 |
| Best CART response | |||
| CR | 7 | 7 | 0 |
| PR | 3 | 3 | 0 |
| No response/stable disease | 2 | 0 | 2 |
| Progressive disease | 1 | 0 | 1 |
| Post-CART therapies | |||
| None | 5 | 5 | 0 |
| HSCT | |||
| During CART-mediated remission | 2 | 2§ | 0 |
| Salvage for R/R disease | 3 | 2§ | 1 |
| Pembrolizumab | 2 | 2 | 0 |
| Salvage systemic therapy | 5 | 3 | 2 |
| Investigational CART therapy | 1 | 1 | 0 |
| Status at last follow-up | |||
| Alive, no disease | 8 | 8 | 0 |
| Alive, with disease | 2 | 2 | 0 |
| Deceased | 3 | 0 | 3 |
| Time after CART at last follow-up median (range), mo | 21.2 (0.9-54) | 25.4 (0.9-54) | 9.8 (3.2-19.7) |
Responders include those with CR and PR. All data are presented as number of patients (unless otherwise specified).
ALC, absolute lymphocyte count; CNS, central nervous system; PMBCL, primary mediastinal BCL.
Out-of-specification product due to 78% product viability.
No grade ≥4 toxicity.
The patient with neurotoxicity had CNS involvement at time of CART.
One patient who underwent preemptive HSCT while in CART-mediated CR subsequently underwent second allogeneic HSCT after experiencing disease relapse.