Baseline demographic and clinical characteristics
| Characteristics . | Pegcetacoplan (n = 35) . | Control, supportive care only∗ (n = 18) . |
|---|---|---|
| Age, mean (range), y | 42.2 (22-67) | 49.1 (20-74) |
| ≥65 y, n (%) | 2 (5.7) | 4 (22.2) |
| Female, n (%) | 16 (45.7) | 8 (44.4) |
| Race, n (%) | ||
| Asian | 23 (65.7) | 16 (88.9) |
| American Indian or Alaska native | 9 (25.7) | 2 (11.1) |
| Black or African American | 2 (5.7) | 0 |
| White | 0 | 0 |
| Other | 1 (2.9) | 0 |
| Clinical characteristics | ||
| Body mass index, kg/m2, mean (SD) | 24.0 (4.4) | 23.1 (2.9) |
| History of aplastic anemia, n (%) | 5 (14.3) | 5 (27.8) |
| Time since PNH diagnosis, median (range), y | 3.4 (0.1-27.0) | 4.7 (0.1-15.1) |
| Transfusion in previous 12 mo, n (%) | 29 (82.9) | 14 (77.8) |
| <4 transfusions, n (%) | 21 (60.0) | 8 (44.4) |
| ≥4 transfusions, n (%) | 14 (40.0) | 10 (55.6) |
| Laboratory measurements, mean (SD) | ||
| Hemoglobin, g/dL† | 9.4 (1.4) | 8.7 (0.8) |
| LDH, U/L‡ | 2151.0 (909.4) | 1945.9 (1003.7) |
| ARC, ×109 cells per L§ | 230.2 (81.0) | 180.3 (109.1) |
| Total bilirubin, μmol/L‖ | 39.4 (20.5) | 35.5 (15.0) |
| Indirect bilirubin, μmol/L¶ | 34.1 (18.5) | 30.5 (13.5) |
| FACIT-Fatigue score# | 36.3 (10.7) | 37.1 (9.3) |
| EORTC QLQ-C30 global health status/QoL score∗∗ | 63.3 (19.7) | 62.0 (15.8) |
| Characteristics . | Pegcetacoplan (n = 35) . | Control, supportive care only∗ (n = 18) . |
|---|---|---|
| Age, mean (range), y | 42.2 (22-67) | 49.1 (20-74) |
| ≥65 y, n (%) | 2 (5.7) | 4 (22.2) |
| Female, n (%) | 16 (45.7) | 8 (44.4) |
| Race, n (%) | ||
| Asian | 23 (65.7) | 16 (88.9) |
| American Indian or Alaska native | 9 (25.7) | 2 (11.1) |
| Black or African American | 2 (5.7) | 0 |
| White | 0 | 0 |
| Other | 1 (2.9) | 0 |
| Clinical characteristics | ||
| Body mass index, kg/m2, mean (SD) | 24.0 (4.4) | 23.1 (2.9) |
| History of aplastic anemia, n (%) | 5 (14.3) | 5 (27.8) |
| Time since PNH diagnosis, median (range), y | 3.4 (0.1-27.0) | 4.7 (0.1-15.1) |
| Transfusion in previous 12 mo, n (%) | 29 (82.9) | 14 (77.8) |
| <4 transfusions, n (%) | 21 (60.0) | 8 (44.4) |
| ≥4 transfusions, n (%) | 14 (40.0) | 10 (55.6) |
| Laboratory measurements, mean (SD) | ||
| Hemoglobin, g/dL† | 9.4 (1.4) | 8.7 (0.8) |
| LDH, U/L‡ | 2151.0 (909.4) | 1945.9 (1003.7) |
| ARC, ×109 cells per L§ | 230.2 (81.0) | 180.3 (109.1) |
| Total bilirubin, μmol/L‖ | 39.4 (20.5) | 35.5 (15.0) |
| Indirect bilirubin, μmol/L¶ | 34.1 (18.5) | 30.5 (13.5) |
| FACIT-Fatigue score# | 36.3 (10.7) | 37.1 (9.3) |
| EORTC QLQ-C30 global health status/QoL score∗∗ | 63.3 (19.7) | 62.0 (15.8) |
SD, standard deviation.
Control group patients received supportive care (eg, transfusions, corticosteroids, and supplements [iron, folate, and vitamin B12]).
Normal reference range: male, 13.6 to 18 g/dL; and female, 12 to 16 g/dL.
Normal reference range: 113 to 226 U/L.
Normal reference range: male, 10 × 109 to 140 × 109 cells per L; and female, 10 × 109 to 120 ×109 cells per L.
Normal reference range: 1.7 to 18.8 μmol/L.
Normal reference range: 1.7 to 15.4 μmol/L.
General population norm: 43.6. Defined by Cella et al.23
General population norm: 75.7. Defined by Hinz et al.24