Patient demographics
| . | Nonbleeding . | Bleeding . | P . |
|---|---|---|---|
| Number of patients | 38 | 19 | — |
| Sex | |||
| Male | 22 | 14 | — |
| Female | 16 | 5 | — |
| Race | |||
| White | 28 | 16 | — |
| Black/African American | 4 | 3 | — |
| Asian | 3 | 0 | — |
| Mixed | 3 | 0 | — |
| Age, y | 4.9 (0.5-25.9) | 10.0 (0.6-29.9) | NS |
| Bodyweight, kg | 21.3 (5.0-105) | 29.1 (5.5-91.6) | NS |
| GFR, mL/min per 1.73 m2 | 175 (21.6-501) | 136 (19.7-894) | NS |
| SCR, mg/dL | 0.4 (0.2-2.2) | 0.34 (0.18-1.6) | NS |
| BIL, mg/dL | 0.7 (1.0-12.5) | 1.2 (0.2-42.2) | NS |
| ALB, mg/dL | 3.2 (2.0-4.2) | 2.7 (2.1-3.4) | <.01 |
| AST, U/L | 62 (13-432) | 38 (14-135) | <.05 |
| ALT, U/L | 39 (14-643) | 34 (11-248) | NS |
| Predose sC5b-9, ng/mL | 217 (107-1035) | 339 (126-1641) | NS |
| RBC transfusion | |||
| Patients, n/total in group | 5 (2/38) | 84 (16/19) | <.0001 |
| Total amount, mL/kg* | 0 (0-113) | 30 (0-151) | <.0001 |
| Transfusion days, n† | 0 (0-7) | 4 (0-34) | <.01 |
| Platelet transfusions | |||
| Patients, n/per total group | 5 (2/38) | 79 (15/19) | <.0001 |
| Total amount, mL/kg* | 0 (0-328) | 48 (0-220) | <.0001 |
| Number of transfusion days† | 0 (0-23) | 7 (0-65) | <.0001 |
| First loading dose | |||
| 300 mg | 6 | 4 | — |
| 600 mg | 23 | 6 | — |
| 900 mg | 9 | 9 | — |
| . | Nonbleeding . | Bleeding . | P . |
|---|---|---|---|
| Number of patients | 38 | 19 | — |
| Sex | |||
| Male | 22 | 14 | — |
| Female | 16 | 5 | — |
| Race | |||
| White | 28 | 16 | — |
| Black/African American | 4 | 3 | — |
| Asian | 3 | 0 | — |
| Mixed | 3 | 0 | — |
| Age, y | 4.9 (0.5-25.9) | 10.0 (0.6-29.9) | NS |
| Bodyweight, kg | 21.3 (5.0-105) | 29.1 (5.5-91.6) | NS |
| GFR, mL/min per 1.73 m2 | 175 (21.6-501) | 136 (19.7-894) | NS |
| SCR, mg/dL | 0.4 (0.2-2.2) | 0.34 (0.18-1.6) | NS |
| BIL, mg/dL | 0.7 (1.0-12.5) | 1.2 (0.2-42.2) | NS |
| ALB, mg/dL | 3.2 (2.0-4.2) | 2.7 (2.1-3.4) | <.01 |
| AST, U/L | 62 (13-432) | 38 (14-135) | <.05 |
| ALT, U/L | 39 (14-643) | 34 (11-248) | NS |
| Predose sC5b-9, ng/mL | 217 (107-1035) | 339 (126-1641) | NS |
| RBC transfusion | |||
| Patients, n/total in group | 5 (2/38) | 84 (16/19) | <.0001 |
| Total amount, mL/kg* | 0 (0-113) | 30 (0-151) | <.0001 |
| Transfusion days, n† | 0 (0-7) | 4 (0-34) | <.01 |
| Platelet transfusions | |||
| Patients, n/per total group | 5 (2/38) | 79 (15/19) | <.0001 |
| Total amount, mL/kg* | 0 (0-328) | 48 (0-220) | <.0001 |
| Number of transfusion days† | 0 (0-23) | 7 (0-65) | <.0001 |
| First loading dose | |||
| 300 mg | 6 | 4 | — |
| 600 mg | 23 | 6 | — |
| 900 mg | 9 | 9 | — |
ALB, albumin; ALT, alanine aminotransferase; AST, aspartate aminotransferase; BIL, serum bilirubin; GFR, glomerular filtration rate adjusted by 1.73 m2 of body surface area; PLTS, platelet; RBC, red blood cell; SCR, serum creatinine.
Total amount, sum of RBC/PLTS transfusions (mg/kg per day) during the first 5 treatment doses.
Number of transfusions, sum of the RBC/PLTS transfusion days during the first 5 treatment doses.