TABLE 1.
PNH Cohort, Patient Characteristics, by Index C5i Therapy
| Variable | Eculizumab(n = 34) | Ravulizumab(n = 52) |
|---|---|---|
| Baseline characteristics | ||
| Age, mean (SD) | 40.9 (12) | 43.7 (13.8) |
| Age group, n (%) | ||
| 18-49 years | 25 (73.5) | 34 (65.4) |
| 50-64 years | 8 (23.5) | 15 (28.9) |
| ≥ 65 years | 1 (2.9) | 3 (5.8) |
| Sex, n (%) | ||
| Female | 18 (52.9) | 29 (55.8) |
| Male | 16 (47.1) | 23 (44.2) |
| Quan-Charlson comorbidity index, mean (SD) | 2.4 (2.7) | 1.5 (2.7) |
| HRU and costs in pre-index period | ||
| Total HRU cost of care, mean (SD) | $263,665 ($208,729) | $240,993 ($153,667) |
| Total HRU cost of care, median | $251,034 | $242,809 |
| Number of inpatient visits, mean (SD) | 2.1 (1.6) | 1.6 (0.8) |
| Number of ED visits, mean (SD) | 1.3 (0.5) | 1.6 (1.2) |
| Number of transfusions, mean (SD) | 7.4 (8.1) | 7.4 (7.0) |
| Pre-index and post-index comorbidities and clinical complications, n (%) | ||
| Aplastic anemia | ||
| Pre-index period | 12 (35.3) | 23 (44.2) |
| Post-index period | 17 (50.0) | 23 (44.2) |
| Anemia | ||
| Pre-index period | 21 (61.8) | 34 (65.4) |
| Post-index period | 23 (67.7) | 34 (65.4) |
| Bleeding | ||
| Pre-index period | 0 (0.0) | 3 (5.8) |
| Post-index period | 3 (8.8) | 3 (5.8) |
| BTH | ||
| Pre-index period | 8 (23.5) | 7 (13.5) |
| Post-index period | 10 (29.4) | 7 (13.5) |
| MDS | ||
| Pre-index period | 4 (11.8) | 4 (7.7) |
| Post-index period | 4 (11.8) | 5 (9.6) |
| Infection | ||
| Pre-index period | 9 (26.5) | 6 (11.5) |
| Post-index period | 12 (35.3) | 14 (26.9) |
| Thrombosis | ||
| Pre-index period | 1 (2.9) | 4 (7.7) |
| Post-index period | 5 (14.7) | 7 (13.5) |
| Dyspnea | ||
| Pre-index period | 12 (35.3) | 9 (17.3) |
| Post-index period | 9 (26.5) | 10 (19.2) |
All costs are shown in US dollars.
BTH = breakthrough hemolysis; C5i = C5 inhibitor; ED = emergency department; HRU = health care resource utilization; MDS = myelodysplastic syndrome; PNH = paroxysmal nocturnal hemoglobinuria.