Table 1.
Patient characteristics and baseline clinical parameters†
| Parameters | TE Cases (n = 57) |
Controls (n = 189) |
|---|---|---|
| Patient Demographics | ||
| Sex, n (%) | ||
| Male | 31 (54.4) | 103 (54.5) |
| Female | 26 (45.6) | 86 (45.5) |
| Region, n (%) | ||
| Europe | 42 (73.7) | 149 (78.8) |
| Asia | 7 (12.3) | 23 (12.2) |
| North America | 6 (10.5) | 15 (7.9) |
| Australia/New Zealand | 2 (3.5) | 2 (1.1) |
| Age at index date, mean (SD) | 46.8 (17.1) | 47.1 (16.0) |
| Years from PNH diagnosis to index date, mean (SD) | 7.4 (8.4) | 7.4 (8.5) |
| History of BMF, n (%) | 39 (68.4) | 149 (78.8) |
| Clinical Parameters | ||
| Percent GPI-negative granulocytes, n (%) | ||
| < 10% | 7 (12.3) | 43 (22.8) |
| ≥ 10% to < 30% | 1 (1.8) | 16 (8.5) |
| ≥ 30% to < 50% | 3 (5.3) | 6 (3.2) |
| ≥ 50% | 17 (29.8) | 25 (13.2) |
| Missing | 29 (50.9) | 99 (52.4) |
| Percent GPI-negative erythrocytes (type II and III), n (%) | ||
| < 10% | 13 (22.8) | 55 (29.1) |
| ≥ 10% | 16 (28.1) | 28 (14.8) |
| Missing | 28 (49.1) | 106 (56.1) |
| LDH ratio (× ULN), n (%) | ||
| < 1.5 | 15 (26.3) | 71 (37.6) |
| ≥ 1.5 | 22 (38.6) | 41 (21.7) |
| Missing | 20 (35.1) | 77 (40.7) |
| HDA‡ | ||
| No | 18 (31.6) | 83 (43.9) |
| Yes | 19 (33.3) | 29 (15.3) |
| Missing | 20 (35.1) | 77 (40.7) |
| LDH ratio (× ULN), n (%) and HDA criteria | ||
| LDH < 1.5 × ULN and no HDA criteria | 4 (7.0) | 27 (14.3) |
| LDH ≥ 1.5 × ULN with 0-1 HDA criterion | 5 (8.8) | 22 (11.6) |
| LDH ≥ 1.5 × ULN with 2–3 HDA criteria | 10 (17.5) | 13 (6.9) |
| LDH ≥ 1.5 × ULN with ≥ 4 HDA criteria | 7 (12.3) | 6 (3.2) |
| Missing | 31 (54.4) | 121 (64.0) |
| History of MAVE, n (%)§ | ||
| No | 42 (73.7) | 169 (89.4) |
| Yes | 14 (24.6) | 20 (10.6) |
| Missing | 1 (1.8) | 0 (0.0) |
| History of non-TE MAVE, n (%)§ | ||
| No | 49 (86.0) | 177 (93.7) |
| Yes | 7 (12.3) | 12 (6.3) |
| Missing | 1 (1.8) | 0 (0.0) |
| History of TE, n (%)§ | ||
| No | 44 (77.2) | 178 (94.2) |
| Yes | 12 (21.1) | 11 (5.8) |
| Missing | 1 (1.8) | 0 (0.0) |
| Recent anticoagulation, n (%)¶ | ||
| No | 15 (26.3) | 63 (33.3) |
| Yes | 27 (47.4) | 21 (11.1) |
| Missing | 15 (26.3) | 105 (55.6) |
†The index date is the first TE event after enrollment in the Registry. Data are based on assessments within 6 months before the TE index date. ‡HDA defined as LDH ratio ≥ 1.5 × ULN, hemoglobin < 10 g/dL, and one of the following symptoms 6 months before and including the index date: abdominal pain, dyspnea, dysphagia, fatigue, hemoglobinuria, or erectile dysfunction. §Assessments based on the entire patient history from PNH diagnosis to index date. ¶Recent is defined as within 6 months before the index date. BMF, bone marrow failure; GPI, glycosylphosphatidylinositol; HDA, high disease activity; LDH, lactate dehydrogenase; MAVE, major adverse vascular event; PNH, paroxysmal nocturnal hemoglobinuria; TE, thromboembolic event; ULN, upper limit of normal