Table 2.
Basal evaluations (V0) and acute safety outcomes (V1-V3) in one patient affected by argininosuccinate lyase (ASL) deficiency and two patients with methylmalonic acidemia treated by intrahepatic injections of human liver stem cells (HLSCs)
| Patient 1 (ASL deficiency) | Patient 2 and 3 (Methylmalonic acidemia) | All patients | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| HLSCs dose | 125,000 cells/g of liver | 250,000 cells/g of liver | ||||||||||
| Visit | V0 | V1 | V2 | V3 | V0 | V1 | V2 | V3 | V0 | V1 | V2 | V3 |
| Clinical parameters | ||||||||||||
| Blood pressure (mmHg) | 91/52 | 90/52 | 75/48 | 72/40 | 73 ± 23/43 ± 23 | 90 ± 0/48 ± 4 | 80 ± 7/52 ± 10 | 85 ± 7/50 ± 0 | 79 ± 19/46 ± 17 | 90 ± 0/49 ± 3 | 75 ± 9/48 ± 10 | 80 ± 9/46 ± 5 |
| Temperature (°C) | 36.1 | 36.3 | 36.7 | 36.0 | 36.3 ± 0.1 | 36.3 ± 0.1 | 36.7 ± 0.7 | 36.2 ± 0.2 | 36.1 ± 0.1 | 36.1 ± 0.6 | 36.7 ± 0.2 | 36.1 ± 0.2 |
| Heart rate | 131 | 135 | 116 | 140 | 98 ± 2 | 100 ± 0 | 97 ± 1 | 99 ± 0 | 121 ± 9 | 137 ± 28 | 116 ± 24 | 139 ± 7 |
| Respiratory rate | 45 | 55 | 47 | 40 | 55 ± 7 | 47 ± 18 | 50 ± 14 | 54 ± 8 | 51 ± 7 | 49 ± 13 | 47 ± 10 | 49 ± 10 |
| Saturation O2(%) | 99 | 100 | 98 | 98 | 98 ± 2 | 100 ± 0 | 97 ± 1 | 99 ± 0 | 98 ± 2 | 100 ± 0 | 98 ± 2 | 99 ± 1 |
| Laboratory tests | ||||||||||||
| Red blood cells (1012/l) | 4.0 | 3.9 | 3.8 | 4.0 | 3.6 ± 0.0 | 4.4 ± 0.4 | 4.4 ± 0.1 | 4.4 ± 0.4 | 3.7 ± 0.2 | 4.3 ± 0.4 | 4.2 ± 0.3 | 4.3 ± 0.3 |
| Hemoglobin (g/dl) | 10.9 | 10.0 | 10.1 | 10.1 | 7.9 ± 0.3 | 10.6 ± 2.4 | 9.9 ± 0.7 | 9.5 ± 0.0 | 8.9 ± 1.7 | 10.4 ± 1.7 | 9.9 ± 0.5 | 9.7 ± 0.3 |
| Platelets (109/l) | 720 | 574 | 761 | 458 | 763 ± 30 | 484 ± 48 | 588 ± 186 | 502 ± 38 | 749 ± 33 | 514 ± 62 | 645 ± 165 | 487 ± 37 |
| White blood cells (109/l) | 12.4 | 19.8 | 12.4 | 11.8 | 8.8 ± 1.6 | 7.1 ± 0.3 | 10.0 ± 3.4 | 9.1 ± 4.6 | 10.0 ± 2.2 | 11.3 ± 7.3 | 10.8 ± 2.7 | 10.0 ± 3.6 |
| INR | 1.1 | 1.0 | 1.0 | 0.9 | 1.0 ± 0.2 | 1.0 ± 0.2 | 1.0 ± 0.2 | 1.0 ± 0.1 | 1.1 ± 0.2 | 1.0 ± 0.1 | 1.0 ± 0.1 | 1.0 ± 0.0 |
| AST (U/l) | 32 | 37 | 65 | 43 | 41 ± 22 | 32 ± 4 | 33 ± 9 | 37 ± 14 | 38 ± 16 | 33 ± 4 | 43 ± 20 | 39 ± 10 |
| ALT (U/l) | 34 | 33 | 39 | 25 | 39 ± 18 | 32 ± 4 | 34 ± 16 | 37 ± 23 | 37 ± 13 | 32 ± 3 | 36 ± 11 | 33 ± 18 |
International normalized ratio (INR, normal range 0.8–1.2); Aspartate aminotransferase (AST, normal range: 20–50 U/l); Alanine aminotransferase (ALT, normal range: 20–50 U/l)