Table II.
Summary of clinical trials utilizing UCB that has been expanded ex vivo.
Type of expansion | Reference | Subjects | Cytokines | Days in culture | Fold-expansion
|
Days to absolute neutrophil count (ANC) >500 | Days to platelets >20 000 | Survival (median length) and aGvHD | |
---|---|---|---|---|---|---|---|---|---|
TNC | CD34+ | ||||||||
Liquid suspension | 16 | n = 37 adults and children | SCF, TPO, G-CSF | 10 | 56 | 4 | 28 | 106 | 32% survival (minimum 17 months), 67% grade II–IV, 40% grade III–IV |
57 | n = 35 adults and children | SCF, TPO, G-CSF | 14 | 23 | 2.3 | 14 | 34 | 48% survival (11 months), 43% grade II–IV, 7% grade III–IV | |
20 | n = 10 adults and children | SCF, FL, IL-6, TPO, TEPA | 21 | 219 | 6 | 30 | 48 | 30% survival (25 months), 44% grade II, no grade III–IV | |
21 | n = 5 adults& children | Notch ligand delta1, SCF, FL, IL-6, TPO, IL-3 | 16 | 660 | 160 | 14 | 83% survival (277 days) | ||
Stromal co-culture | 22 | n = 6 adults and children | SCF, TPO, G-CSF | 14 | 12 | 12 | 14.5 | 30 | 83% survival (12 months), 33% grade II, no grade III–IV |
Continuous perfusion system | 18 | n = 27 children and a few adults | PIXY321, FL, EPO | 12 | 2.4 | 0.5 | 22 | 71 | 39% survival (41 months)n 36% grade II–IV, 22% grade III–IV |
17 | n = 2 adults | PIXY321, FL, EPO | 12 | 2.2 | 1.6, second did not expand | 28 | 56 | 100% survival (13 months), no |