Table 1.
Characteristics of patients and grafts on studies with omidubicel and UM171 expanded UCB.
| Variable | Omidubicel | UM171 |
|---|---|---|
| Trial | Phase 3 | Phase 1–2 |
| Year of publication | Oct 2021 | Feb 2022 |
| FDA approval | April 2023 | – |
| No. CBU | 1 expanded UCB (n = 52) versus Standard UCB (n = 56, single or d UCB) | 1 expanded UCB plus 1 unexpanded UCB (n = 5) 1 expanded UCB (n = 22) |
| HLA match | >4 of 6 | >4 of 6 |
| CBU requirement before expansion (median dose) | ||
| TNC (×107/kg) | 3.0 (1.6–7.3) | 2.92 (1.45–5.91) |
| CD34 (×108/kg) | 0.14 (0.09–0.4) | 1.44 (0.66–5.91) |
| Expansion culture duration (days) | 21 ±2 | 7 |
| Post expansion (median dose) | ||
| CD34 (×106/kg) | 9.0 (2.1–47.6) | 2.87 (1.91–3.96) |
| CD34 expansion, fold | 130 (1.91–3.96) | 28 (19–35) |
| Hematologic malignancies, high risk | MDS, int-2 to high AML, CR1/CR2 ALL, CR1/CR2/CR3 CLL & Lymphomas |
MDS, MPN Acute Leukemia, CR1/CR2/not in CR, CLL & Lymphomas |
| Conditioning | Myeloablative | Myeloablative |
| Engraftment | ||
| ANC (days, range) | 10 (8–13) | 18 (12.5–20) |
| Platelets (days, range) | 37 (33–42) | 42 (35–47) |
| Acute GVHD | ||
| Grade 2–3 | 56% at D100 | 64% at 1 year |
| Grade 3–4 | 14% at D100 | 0% at 1 year |
| Chronic GVHD | ||
| All grade | 35% at 1 year | 17% at 1 year |
| Moderate-severe | 27% at 1 year | 0% at 1 year |
| Median F/U (months) | 10 (1–19) | 18 (12–22) |
| TRM | 11% versus 24% at 210 days | 5% at 1 year |
| Relapse | 25% versus 17% (p = 0.32) At 15 months |
21% (9–51) At 12 months |
| GRFS at 1 year | 36% versus 45% (p = 0.56) | 64% (43–86) |
ALL, acute lymphocytic leukemia; AML, acute myelogenous leukemia; ANC, absolute neutrophil count; CLL, chronic lymphocytic leukemia; F/U, follow-up; FDA, U.S. Food & Drug Administration; GVHD, graft-versus-host disease; MDS, myelodysplastic syndrome; MPN, myeloproliferative neoplasm; TNC, total nucleated cell; TRM, transplant related mortality; UCB, umbilical cord blood.