Table 2. . Disease and management characteristics as reported by adult patients and by caregivers on behalf of pediatric patients.
| Demographic variable | Adult patients (n = 50) | Pediatric patients (n = 16) |
|---|---|---|
| Years since diagnosis Mean (SD) Range |
6.8 (7.5) 1–48 |
6.1 (3.8) 1–14 |
| Trigger Infection Severe hypertension Autoimmune disease Pregnancy or childbirth None Other† Unsure |
11 (22.0) 2 (4.0) 1 (2.0) 9 (18.0) 2 (4.0) 12 (24.0) 13 (26.0) |
6 (37.5) 0 (0.0) 0 (0.0) 0 (0.0) 4 (25.0) 2 (12.5) 4 (25.0) |
| Length of eculizumab treatment, months Mean (SD) Range |
46.9 (33.4) 2–133 |
46.5 (32.1) 3–119 |
| Site of last eculizumab treatment Hospital (inpatient) Hospital (outpatient) Clinic Home |
0 (0.0) 10 (20.0) 24 (48.0) 16 (32.0) |
1 (6.3) 6 (37.5) 4 (25.0) 5 (31.3) |
| Length of ravulizumab treatment, months‡ Mean (SD) Range |
12.9 (6.0) 0–25 |
13.6 (5.7) 4–27 |
| Site of last ravulizumab treatment Hospital (inpatient) Hospital (outpatient) Clinic Home |
0 (0.0) 8 (16.0) 28 (56.0) 14 (28.0) |
1 (6.3) 6 (37.5) 4 (25.0) 5 (31.3) |
| Current ravulizumab formulation 100 mg/ml 10 mg/ml Unsure |
39 (78.0) 8 (16.0) 3 (6.0) |
12 (75.0) 2 (12.5) 2 (12.5) |
| Venous access port previously implanted for eculizumab Yes No |
17 (34.0) 33 (66.0) |
10 (62.5) 6 (37.5) |
| Venous access port currently implanted for ravulizumab Yes No |
15 (30.0) 35 (70.0) |
8 (50.0) 8 (50.0) |
†
Participant free-text responses mentioned medication use, transplantation, surgery, pulmonary hemorrhages, pneumonia, pregnancy, infections, stress and vaccination.
‡
Length of treatment was calculated using the difference between the dates of the first and last treatments as reported by participants. The minimum value of 0 months on ravulizumab may be caused by a participant adding the same date for both responses.
SD: Standard deviation.