Baseline characteristics
Characteristic . | Danicopan-danicopan (n = 57) . | Placebo-danicopan (n = 29) . |
---|---|---|
Age, mean (min-max), y | 52.8 (20-82) | 52.9 (29-77) |
Sex, n (%) | ||
Female | 34 (59.6) | 20 (69.0) |
Male | 23 (40.4) | 9 (31.0) |
Hb, mean (SD), g/dL | 7.7 (0.9) | 7.9 (1.0) |
ARC, mean (SD), ×109/L | 247.6 (97.2) | 222.7 (115.4) |
LDH∗, mean (SD), U/L | 304.0 (123.6) | 286.4 (93.1) |
FACIT-Fatigue scores, mean (SD) | 34.0 (11.3) | 31.7 (11.0) |
Participants requiring transfusion instances 6 months before screening, n (%) | 56 (98.2) | 28 (96.6) |
Number of transfusion instances 6 months before screening, median (range) | 2.0 (0-8) | 3.0 (0-8) |
C5 inhibitor, n (%) | ||
Ravulizumab | 36 (63.2) | 15 (51.7) |
Eculizumab | 21 (36.8) | 14 (48.3) |
Duration of C5 inhibitor therapy†, mean (SD), y | 5.1 (3.6) | 6.1 (4.2) |
Duration of disease‡, mean (SD), y | 10.0 (9.7) | 11.0 (9.5) |
Proportion of type III PNH RBC population§, mean (SD), % | 57.6 (26.1) | 53.7 (28.2) |
C3 deposition on type III PNH RBCs§, mean (SD), % | 33.4 (18.2) | 30.4 (16.0) |
Medical history of aplastic anemia||, n (%) | 18 (31.6) | 8 (27.6) |
Medical history of myelodysplastic syndrome||, n (%) | 2 (3.5) | 3 (10.3) |
Characteristic . | Danicopan-danicopan (n = 57) . | Placebo-danicopan (n = 29) . |
---|---|---|
Age, mean (min-max), y | 52.8 (20-82) | 52.9 (29-77) |
Sex, n (%) | ||
Female | 34 (59.6) | 20 (69.0) |
Male | 23 (40.4) | 9 (31.0) |
Hb, mean (SD), g/dL | 7.7 (0.9) | 7.9 (1.0) |
ARC, mean (SD), ×109/L | 247.6 (97.2) | 222.7 (115.4) |
LDH∗, mean (SD), U/L | 304.0 (123.6) | 286.4 (93.1) |
FACIT-Fatigue scores, mean (SD) | 34.0 (11.3) | 31.7 (11.0) |
Participants requiring transfusion instances 6 months before screening, n (%) | 56 (98.2) | 28 (96.6) |
Number of transfusion instances 6 months before screening, median (range) | 2.0 (0-8) | 3.0 (0-8) |
C5 inhibitor, n (%) | ||
Ravulizumab | 36 (63.2) | 15 (51.7) |
Eculizumab | 21 (36.8) | 14 (48.3) |
Duration of C5 inhibitor therapy†, mean (SD), y | 5.1 (3.6) | 6.1 (4.2) |
Duration of disease‡, mean (SD), y | 10.0 (9.7) | 11.0 (9.5) |
Proportion of type III PNH RBC population§, mean (SD), % | 57.6 (26.1) | 53.7 (28.2) |
C3 deposition on type III PNH RBCs§, mean (SD), % | 33.4 (18.2) | 30.4 (16.0) |
Medical history of aplastic anemia||, n (%) | 18 (31.6) | 8 (27.6) |
Medical history of myelodysplastic syndrome||, n (%) | 2 (3.5) | 3 (10.3) |