TABLE 3.
Clinical trials for ex vivo manipulation of UCB to transplant
| Expansion approach | Clinical trial staging | Expanded TNC, Median (range), ×107/kg | Expanded CD34+, Median (range), ×105/kg | Neutrophil engraftment rate | Neutrophil engraftment time, median (range), days | Platelet engraftment time median (range), days | |
|---|---|---|---|---|---|---|---|
| Delaney et al 79 | Notch ligand | Phase I (n = 10) | 4.6 (0.6‐9.1) | 60 (9.3‐130) | N.R. | 16 (7‐34) | N.R. |
| Horwitz et al 80 | NiCord dUCBT | Phase I (n = 11) | 2.5 (1.7‐3.8) | 35 (9‐183) | 42d 90.9% | 13 (7‐26) | 33 (26‐49) |
| Horwitz et al 81 | NiCord sUCBT | Phase I/II (n = 36) | 4.9 (2.0‐13.3) | 63 (14‐149) | 42d 94% | 11.5 (9‐14) a | 34 (32‐42) a |
| de Lima et al 82 | Copper chelator (StemEx) | Phase I/II (n = 10) | 0.9 (0.1‐29.8) | 1.2 (0.16‐45.04) | 46d 90% | 30 (16‐46) | 48 (35‐105) |
| Stiff et al 83 | Copper chelator (StemEx) | N.R. (n = 101) | 2.2 (N.R.) | 9.26 (0.83‐117.56) | 91.9% | 21 (18.4‐23.5) a | 54 (43.3‐61.9) a |
| Wagner et al 84 | StemRegenin‐1 (SR1) | Phase I/II (n = 17) | 5 (1‐12) | 175 (14‐483) | 100% | 15 (6‐30) | 49 (28‐136) |
| Cohen et al 85 | UM171 | Phase I/II (n = 22) | N.R. | 28.75 (7.89‐54.58) | 100% | 18 (12.5‐20.0) b | 42 (35‐47) b |
| de Lima et al 72 | MSC | N.R. (n = 24) | 5.84 (0.03‐14.37) | 9.5 (16‐93.4) | 42d 96% | 15 (9‐42) | 42 (15‐62) |
| Mehta et al 86 | MPC | N.R. (n = 27) | 5.7 (1.35‐11.8) | 16 (0.4‐53) | 26d 78% | 12 (1‐28) | 31 (9‐52) |
a
95% confidence interval.
b
Interquartile range.
Abbreviations: dUCBT, double‐unit UCBT; MPC, mesenchymal precursor cell; MSC, mesenchymal stromal cell; N.R., not reported; sUCBT, single‐unit UCBT; TNC, total nucleated cell; UCB, umbilical cord blood.