Table 4.
Summary of investigator-assessed efficacy parameters by histology
| Parameter | B-ALL (n = 25) | T-ALL (n = 19) | LL (n = 3) | All patientsa (N = 47) | Pediatrica (n = 12) |
|---|---|---|---|---|---|
| Responseb, n (%) | 16 (64.0) | 10 (52.6) | 2 (66.7) | 28 (59.6) | 9 (75.0) |
| CR rate (CR/CRi/CRp) | 3 (12.0) | 0 | 0 | 3 (6.4) | 1 (8.3) |
| PR | 2 (8.0) | 6 (31.6) | 0 | 8 (17.0) | 0 |
| SD | 4 (16.0) | 3 (15.8) | 1 (33.3) | 8 (17.0) | 2 (16.7) |
| PD | |||||
|
| |||||
| Patients with ALL and morphologic CR at baseline, n | n = 1 | n = 4 | NA | n = 5 | n = 1 |
| Response, n (%) | |||||
| CR rate (CR/CRi/CRp) | 0 | 3 (75.0) | 3 (60.0) | 1 (100) | |
| SD | 0 | 1 (25.0) | 1 (20.0) | 0 | |
| NEc | 1 (100) | 0 | 1 (20.0) | 0 | |
|
| |||||
| DORd in all responders | |||||
| n | 19 | 10 | 2 | 31 | 10 |
| Median (95% CI), mo | 9.1 (1.4–14.6) | 4.2 (0.8–12.3) | NE (NE–NE) | 4.2 (2.3–11.5) | 3.5 (0.7–3.5) |
|
| |||||
| OS | |||||
| Median (95% CI), mo | 9.7 (4.0–15.7) | 6.6 (3.2–12.5) | NE (2.0–NE) | 7.8 (4.0–12.5) | NE (2.0–NE) |
| 12-month (95% CI), % | 33.8 (13.7–55.2) | 29.7 (10.4–52.2) | 66.7 (5.4–94.5) | 35.6 (20.9–50.7) | 60.8 (25.0–83.6) |
|
| |||||
| Bone marrow MRD, n (%) | |||||
| MRD negative (<10−4) | 9 (36.0) | 6 (31.6) | 1 (33.3) | 16 (34.0) | 6 (50.0) |
| MRD positive | 10 (40.0) | 3 (15.8) | 1 (33.3) | 14 (29.8) | 5 (41.7) |
| Othere | 6 (24.0) | 10 (52.6) | 1 (33.3) | 17 (36.2) | 1 (8.3) |
|
| |||||
| Proceeded to CAR T-cell therapy or HCT, n (%)f | 8 (32.0) | 3 (15.8) | 2 (66.7) | 13 (27.7) | 7 (58.3) |
Includes all patients on study, including those with LL and morphologic CR at baseline.
Includes patients with known bone marrow blasts at baseline ≥5%. Patients with missing baseline blasts were excluded by immunophenotype but included in the overall study population.
Patient was not evaluable for disease assessment (PD occurred prior to start of chemotherapy).
Patients who went to CAR T-cell therapy or transplant were censored at last tumor assessment date.
MRD assessment was missing or not evaluated (e.g., nonresponders).
Only includes patients who received subsequent transplant or CAR T-cell therapy.
Abbreviations: B-ALL, B-cell acute lymphoblastic leukemia; CAR T, chimeric antigen receptor T cell; CR, complete remission; CRi, complete remission with incomplete marrow recovery; CRp, complete remission without platelet recovery; DOR, duration of response; HCT, hematopoietic cell transplantation; LL, lymphoblastic lymphoma; MRD, minimal residual disease; NA, not applicable; NE, not evaluable; OS, overall survival; PD, progressive disease; PR, partial response; SD, stable disease; T-ALL, T-cell acute lymphoblastic leukemia.