Table 3.
Efficacy of ARI-0001 Cells in Patients with Acute Lymphoblastic Leukemia
| Population | n | MRD-CRR at day +28, rate (95% CI) | MRD-CRR at day +100, rate (95% CI) | PFS Median (95% CI) 1-year rate (95% CI) | DOR Median (95% CI) 1-year rate (95% CI) | OS Median (95% CI) 1-year rate (95% CI) |
|---|---|---|---|---|---|---|
| Total | 38 | 84.2 (69–94) | 71.1 (54–85) | 12.0 mo (4.2–20.2) 47% (27-67%) | 14.8 mo (6.0–NA) 59% (34%–83%) | 20.2 mo (10.4–NA) 69% (49%–88%) |
| Cohorts 1 and 2 (single dose) | 15 | 73.3 (45–92) | 66.7 (38–88) | 17.6 mo (0.3–NA) 51% (25%–77%) | 15.7 (3.6–NA) 70% (42%–98%) | 20.2 mo (0.3–NA) 65.5% (41%–90%) |
| Cohort 3 (fractionated) | 23 | 91.3 (72–99) | 73.9 (52–90) | 12.0 mo (4.2–14.5) 39.5% (4%–75%) | 8.7 mo (2.0–NA) NA (NA–NA) | 14.5 mo (6.8–14.5) 62% (24%–100%) |
| Age groups | ||||||
| ≤18 years | 11 | 81.8 (48–98) | 54.5 (23–83) | 18.1 mo (14.5–NA) 82% (59%–100%) | NA mo (14.8–NA) 100% (100%–100%) | NR (7.1–NA) 78% (50%–100%) |
| >18 years | 27 | 85.2 (66–96) | 77.8 (58–91) | 9.4 mo (3.3–20.2) 34% (12%–57%) | 8.7 mo (3.9–16.6) 46% (18%–75%) | 20.2 mo (12.8–NA) 64.5% (40%–89%) |
| ≤25 years | 19 | 78.9 (54–94) | 63.2 (38–84) | 17.6 mo (4.2–20.2) 64% (40%–89%) | 14.8 mo (6.0–NR) 79% (51%–100%) | 20.2 mo (14.5–NA) 82% (63%–100%) |
| >25 years | 19 | 89.4 (67–99) | 78.9 (54–94) | 7.2 mo (3.2–NA) 25% (0%–52%) | 8.7 mo (2.0–NR) 34% (0%–70%) | NR (6.9–NA) 50.5% (18%–84%) |
MRD-CRR, complete response rate with negative measurable residual disease; PFS, progression-free survival; DOR, duration of response; OS, overall survival; mo, months; NA, not available.