Patient disposition
| Variable . | Eculizumab-naive (n = 5) . | Eculizumab-experienced (n = 8) . | Total (N = 13) . |
|---|---|---|---|
| Sex, n (%) | |||
| Male | 4 (80.0) | 1 (12.5) | 5 (38.5) |
| Female | 1 (20.0) | 7 (87.5) | 8 (61.5) |
| Ravulizumab treatment duration, d, mean ± SD (range) | 883.4 ± 102.7 (700-937) | 1040.4 ± 381.8 (369-1610) | 980.0 ± 308.0 (369-1610) |
| Age at first ravulizumab infusion, y, mean ± SD (range) | 14.4 ± 2.2 (11.0-17.0) | 14.4 ± 3.1 (9.0-17.0) | 14.4 ± 2.7 (9.0-17.0) |
| Age category, y, n (%) | |||
| 0-12 | 1 (20.0) | 1 (12.5) | 2 (15.4) |
| >12 | 4 (80.0) | 7 (87.5) | 11 (84.6) |
| Race, n (%) | |||
| Caucasian | 5 (100) | 3 (37.5) | 8 (61.5) |
| African American | 0 (0) | 2 (25.0) | 2 (15.4) |
| Not reported | 0 (0) | 2 (25.0) | 2 (15.4) |
| Other | 0 (0) | 1 (12.5) | 1 (7.7) |
| Body weight, kg, mean ± SD (range) | 56.3 ± 11.6 (39.5-72.0) | 56.3 ± 12.2 (36.7-69.0) | 56.3 ± 11.5 (36.7-72.0) |
| Weight category, kg, n (%) | |||
| ≥30 to <40 | 1 (20.0) | 1 (12.5) | 2 (15.4) |
| ≥40 to <60 | 3 (60.0) | 4 (50.0) | 7 (53.8) |
| ≥60 to <100 | 1 (20.0) | 3 (37.5) | 4 (30.8) |
| Height, cm, mean ± SD (range) | 163.4 ± 11.8 (143.0-171.0) | 161.0 ± 9.4 (146.0-176.2) | 161.9 ± 9.9 (143.0-176.2) |
| Presenting symptoms, n (%) | |||
| Any PNH symptoms before informed consent | 5 (100) | 7 (87.5) | 12 (92.3) |
| Fatigue/asthenia | 5 (100) | 7 (87.5) | 12 (92.3) |
| Abdominal pain | 3 (60.0) | 5 (62.5) | 8 (61.5) |
| Red/dark urine | 4 (80.0) | 4 (50.0) | 8 (61.5) |
| Jaundice | 4 (80.0) | 3 (37.5) | 7 (53.8) |
| CNS-related symptoms∗ | 2 (40.0) | 4 (50.0) | 6 (46.2) |
| Back or flank pain | 0 (0) | 3 (37.5) | 3 (23.1) |
| Chest pain | 0 (0) | 2 (25.0) | 2 (15.4) |
| Dysphagia | 0 (0) | 1 (12.5) | 1 (7.7) |
| Erectile dysfunction | 0 (0) | 1 (12.5) | 1 (7.7) |
| Leg pain | 0 (0) | 1 (12.5) | 1 (7.7) |
| Dyspnea | 0 (0) | 1 (12.5) | 1 (7.7) |
| Other | 0 (0) | 1 (12.5) | 1 (7.7) |
| Age at PNH diagnosis, y, mean ± SD (range) | 13.8 ± 2.4 (11.0-17.0) | 12.3 ± 3.1 (7.0-16.0) | |
| Time from PNH diagnosis to informed consent, months, mean ± SD (range) | 8.9 ± 1.4 (0-39.6) | 24.1 ± 1.0 (13.2-45.6) | |
| History of pRBC/whole blood transfusion† , n (%) | 2 (40.0) | 2 (25.0) | |
| Units of pRBC/whole blood transfusion, total, mean ± SD (range) | 14.0 7.0 ± 5.7 (3.0-11.0) | 2.0 2.0 (2.0-2.0) | |
| History of aplastic anemia, n (%) | 3 (60.0) | 1 (12.5) | |
| LDH at baseline (U/L), mean ± SD (range)‡ | 957.0 ± 757.2 (444.0-2269.7) | 262.8 ± 106.0 (140.5-487.0) | |
| PNH clone size, mean ± SD (range) | |||
| RBC type II | 18.7 ± 19.5 (0.7-41.4)§ | 10.6 ± 16.4 (0.6-42.6)‖ | |
| RBC type III | 19.2 ± 12.9 (6.2-39.9) | 54.5 ± 22.2 (20.6-80.8) | |
| Total RBC | 38.8 ± 31.5 (6.9-68.1)§ | 65.7 ± 22.7 (21.2-85.4)‖ | |
| Granulocytes | 68.1 ± 26.4 (36.8-99.0) | 82.9 ± 26.0 (20.3-97.6) | |
| Monocytes | 75.2 ± 24.0 (34.9-98.9) | 91.9 ± 5.5 (81.3-97.7) |
| Variable . | Eculizumab-naive (n = 5) . | Eculizumab-experienced (n = 8) . | Total (N = 13) . |
|---|---|---|---|
| Sex, n (%) | |||
| Male | 4 (80.0) | 1 (12.5) | 5 (38.5) |
| Female | 1 (20.0) | 7 (87.5) | 8 (61.5) |
| Ravulizumab treatment duration, d, mean ± SD (range) | 883.4 ± 102.7 (700-937) | 1040.4 ± 381.8 (369-1610) | 980.0 ± 308.0 (369-1610) |
| Age at first ravulizumab infusion, y, mean ± SD (range) | 14.4 ± 2.2 (11.0-17.0) | 14.4 ± 3.1 (9.0-17.0) | 14.4 ± 2.7 (9.0-17.0) |
| Age category, y, n (%) | |||
| 0-12 | 1 (20.0) | 1 (12.5) | 2 (15.4) |
| >12 | 4 (80.0) | 7 (87.5) | 11 (84.6) |
| Race, n (%) | |||
| Caucasian | 5 (100) | 3 (37.5) | 8 (61.5) |
| African American | 0 (0) | 2 (25.0) | 2 (15.4) |
| Not reported | 0 (0) | 2 (25.0) | 2 (15.4) |
| Other | 0 (0) | 1 (12.5) | 1 (7.7) |
| Body weight, kg, mean ± SD (range) | 56.3 ± 11.6 (39.5-72.0) | 56.3 ± 12.2 (36.7-69.0) | 56.3 ± 11.5 (36.7-72.0) |
| Weight category, kg, n (%) | |||
| ≥30 to <40 | 1 (20.0) | 1 (12.5) | 2 (15.4) |
| ≥40 to <60 | 3 (60.0) | 4 (50.0) | 7 (53.8) |
| ≥60 to <100 | 1 (20.0) | 3 (37.5) | 4 (30.8) |
| Height, cm, mean ± SD (range) | 163.4 ± 11.8 (143.0-171.0) | 161.0 ± 9.4 (146.0-176.2) | 161.9 ± 9.9 (143.0-176.2) |
| Presenting symptoms, n (%) | |||
| Any PNH symptoms before informed consent | 5 (100) | 7 (87.5) | 12 (92.3) |
| Fatigue/asthenia | 5 (100) | 7 (87.5) | 12 (92.3) |
| Abdominal pain | 3 (60.0) | 5 (62.5) | 8 (61.5) |
| Red/dark urine | 4 (80.0) | 4 (50.0) | 8 (61.5) |
| Jaundice | 4 (80.0) | 3 (37.5) | 7 (53.8) |
| CNS-related symptoms∗ | 2 (40.0) | 4 (50.0) | 6 (46.2) |
| Back or flank pain | 0 (0) | 3 (37.5) | 3 (23.1) |
| Chest pain | 0 (0) | 2 (25.0) | 2 (15.4) |
| Dysphagia | 0 (0) | 1 (12.5) | 1 (7.7) |
| Erectile dysfunction | 0 (0) | 1 (12.5) | 1 (7.7) |
| Leg pain | 0 (0) | 1 (12.5) | 1 (7.7) |
| Dyspnea | 0 (0) | 1 (12.5) | 1 (7.7) |
| Other | 0 (0) | 1 (12.5) | 1 (7.7) |
| Age at PNH diagnosis, y, mean ± SD (range) | 13.8 ± 2.4 (11.0-17.0) | 12.3 ± 3.1 (7.0-16.0) | |
| Time from PNH diagnosis to informed consent, months, mean ± SD (range) | 8.9 ± 1.4 (0-39.6) | 24.1 ± 1.0 (13.2-45.6) | |
| History of pRBC/whole blood transfusion† , n (%) | 2 (40.0) | 2 (25.0) | |
| Units of pRBC/whole blood transfusion, total, mean ± SD (range) | 14.0 7.0 ± 5.7 (3.0-11.0) | 2.0 2.0 (2.0-2.0) | |
| History of aplastic anemia, n (%) | 3 (60.0) | 1 (12.5) | |
| LDH at baseline (U/L), mean ± SD (range)‡ | 957.0 ± 757.2 (444.0-2269.7) | 262.8 ± 106.0 (140.5-487.0) | |
| PNH clone size, mean ± SD (range) | |||
| RBC type II | 18.7 ± 19.5 (0.7-41.4)§ | 10.6 ± 16.4 (0.6-42.6)‖ | |
| RBC type III | 19.2 ± 12.9 (6.2-39.9) | 54.5 ± 22.2 (20.6-80.8) | |
| Total RBC | 38.8 ± 31.5 (6.9-68.1)§ | 65.7 ± 22.7 (21.2-85.4)‖ | |
| Granulocytes | 68.1 ± 26.4 (36.8-99.0) | 82.9 ± 26.0 (20.3-97.6) | |
| Monocytes | 75.2 ± 24.0 (34.9-98.9) | 91.9 ± 5.5 (81.3-97.7) |
CNS, central nervous system; LDH, lactate dehydrogenase; PNH, paroxysmal nocturnal hemoglobinuria; pRBC, packed red blood cell; RBC, red blood cell; SD, standard deviation; y, years.
For example headache, dizziness, or difficulty concentrating.
In the 12 months before first dose of ravulizumab.
There are multiple LDH normal ranges depending on pediatric age and sex (100-220, 100-242, 100-275, 120-290 and 140-280).
n = 4.
n = 6.