Patient demographics and baseline characteristics (full analysis set)
| Characteristics . | Iptacopan 25 mg/100 mg, n = 7 . | Iptacopan 50 mg/200 mg, n = 6 . | Overall, n = 13 . |
|---|---|---|---|
| Age (y), median (range) | 30 (20-55) | 36 (34-62) | 35 (20-62) |
| Sex, male, n | 5 | 1 | 6 |
| Race, Asian, n | 7 | 6 | 13 |
| BMI, median (range) | 22 (16-27) | 23 (18-29) | 22 (16-29) |
| RBC transfusions in prior year, mean; median (range) | 7; 2.0 (0-19) | 5; 3.5 (0-13) | 6; 3.0 (0-19) |
| Anti-C5 naive, n (%) | 7 (100) | 5 (83) | 12 (92) |
| LDH level (U/L), mean; median (range) | 2228.0; 1686 (1008-3761) | 1946.0; 1737 (1212-2832) | 2097.8; 1686 (1008-3761) |
| Hb level (g/L), n*; mean; median (range) | 7; 90.3; 93 (70-107) | 5; 79.4; 73 (68-96) | 12; 85.8; 87 (68-107) |
| Free Hb level (IU/mL), n; mean; median (range) | 5; 43.9; 36.2 (31.6-82.5) | 4; 24.28; 25.2 (14.2-32.6) | 9; 35.19; 32.6 (14.2-82.5) |
| Red cell count (×1012/L), mean; median (range) | 3.14; 3.1 (2.4-3.8) | 2.63; 2.5 (1.9-3.7) | 2.91; 3.1 (1.9-3.8) |
| Reticulocyte level (×109/L), mean; median (range) | 205.4; 177 (136-284) | 200.8; 215 (30-352)† | 203.3; 209 (30-352)† |
| Reticulocyte/erythrocyte ratio (%), mean; median (range) | 6.7; 6.5 (4.2-10) | 8.35; 7.1 (1.2-18.9) | 7.46; 6.5 (1.2-18.9) |
| Platelet count (×109/L), mean; median (range) | 195.0; 194 (131-318) | 158.5; 180 (60-199) | 178.2; 188 (60-318) |
| Haptoglobin (g/L), mean; median (range)‡ | 0.05; 0.05 (0.05-0.05) | 0.05; 0.05 (0.05-0.05) | 0.05; 0.05 (0.05-0.05) |
| Total bilirubin (µmol/L), mean; median (range) | 32.4; 27 (24-48) | 32.3; 29 (20-51) | 32.4; 27 (20-51) |
| D-dimer (mg FEU/L), mean; median (range) | 0.380; 0.31 (0.24-0.88) | 0.735; 0.78 (0.40-1.14) | 0.544; 0.40 (0.24-1.14) |
| Characteristics . | Iptacopan 25 mg/100 mg, n = 7 . | Iptacopan 50 mg/200 mg, n = 6 . | Overall, n = 13 . |
|---|---|---|---|
| Age (y), median (range) | 30 (20-55) | 36 (34-62) | 35 (20-62) |
| Sex, male, n | 5 | 1 | 6 |
| Race, Asian, n | 7 | 6 | 13 |
| BMI, median (range) | 22 (16-27) | 23 (18-29) | 22 (16-29) |
| RBC transfusions in prior year, mean; median (range) | 7; 2.0 (0-19) | 5; 3.5 (0-13) | 6; 3.0 (0-19) |
| Anti-C5 naive, n (%) | 7 (100) | 5 (83) | 12 (92) |
| LDH level (U/L), mean; median (range) | 2228.0; 1686 (1008-3761) | 1946.0; 1737 (1212-2832) | 2097.8; 1686 (1008-3761) |
| Hb level (g/L), n*; mean; median (range) | 7; 90.3; 93 (70-107) | 5; 79.4; 73 (68-96) | 12; 85.8; 87 (68-107) |
| Free Hb level (IU/mL), n; mean; median (range) | 5; 43.9; 36.2 (31.6-82.5) | 4; 24.28; 25.2 (14.2-32.6) | 9; 35.19; 32.6 (14.2-82.5) |
| Red cell count (×1012/L), mean; median (range) | 3.14; 3.1 (2.4-3.8) | 2.63; 2.5 (1.9-3.7) | 2.91; 3.1 (1.9-3.8) |
| Reticulocyte level (×109/L), mean; median (range) | 205.4; 177 (136-284) | 200.8; 215 (30-352)† | 203.3; 209 (30-352)† |
| Reticulocyte/erythrocyte ratio (%), mean; median (range) | 6.7; 6.5 (4.2-10) | 8.35; 7.1 (1.2-18.9) | 7.46; 6.5 (1.2-18.9) |
| Platelet count (×109/L), mean; median (range) | 195.0; 194 (131-318) | 158.5; 180 (60-199) | 178.2; 188 (60-318) |
| Haptoglobin (g/L), mean; median (range)‡ | 0.05; 0.05 (0.05-0.05) | 0.05; 0.05 (0.05-0.05) | 0.05; 0.05 (0.05-0.05) |
| Total bilirubin (µmol/L), mean; median (range) | 32.4; 27 (24-48) | 32.3; 29 (20-51) | 32.4; 27 (20-51) |
| D-dimer (mg FEU/L), mean; median (range) | 0.380; 0.31 (0.24-0.88) | 0.735; 0.78 (0.40-1.14) | 0.544; 0.40 (0.24-1.14) |
The values at “baseline” visit are presented.
BMI, body mass index.
Patient 6 had Hb levels of 107 g/L at the baseline visit on study day −18 but was deemed eligible based on Hb levels of 99 g/L at a repeat unscheduled assessment on study day −8.
Patient 8, who had preexisting MDS with multilineage dysplasia, was enrolled despite significant reticulocytopenia (30 × 109/L) at baseline but was allowed to remain on study despite this protocol deviation.
All values are below the normal range.