Patient demographics and features at diagnosis and the start of eculizumab treatment (n = 79 unless otherwise stated)
| Demographics and features . | Value . |
|---|---|
| Presenting features | |
| Male, no. (%) | 40 (51) |
| Age at diagnosis, y, median (range) | 37 (12-79) |
| Documented history of AA/MDS,* no. (%) | 24 (30) |
| Hemoglobinuria, no. (%) | 50 (63) |
| Abdominal pain, no. (%) | 24 (30) |
| Dysphagia, no. (%) | 9 (11) |
| Thrombosis, no. (%) | 4 (5) |
| Anemia (n = 73),† no. (%) | 69 (95) |
| LDH,‡ IU/L, median (range) | 2872 (587-10 300) |
| Thromboses before eculizumab (n) | |
| Venous | |
| Budd-Chiari | 12 |
| Mesenteric | 4 |
| Cerebral | 3 |
| Pulmonary embolus | 3 |
| Deep vein | 4 |
| Other | 3 |
| Arterial | |
| Stroke | 3 |
| Myocardial infarction | 2 |
| At start of eculizumab | |
| Age, y, median (range) | 46 (14-84) |
| LDH level, IU/L, median (range) | 2872 (587-10 300) |
| Neutrophils, ×109/L, median (range) | 2.2 (0.6-11.9) |
| Platelets, ×109/L, median (range) | 149 (11-507) |
| Reticulocytes (absolute), ×109/L, median (range) | 171 (57-415) |
| PNH clone sizes, % (range) | |
| Granulocyte | 96.4 (41.8-100) |
| Erythrocte | 34.0 (2.9-100) |
| Type II erythrocyte§ | 3.8 (0-77.4) |
| Type III erythrocyte§ | 25.0 (2.4-79.6) |
| Treatment at start of eculizumab, no. (%) | |
| Anticoagulation | 46 (58) |
| Ciclosporin | 11 (14) |
| Androgens | 2 (3) |
| No transfusion support‖ | 4 (5) |
| Demographics and features . | Value . |
|---|---|
| Presenting features | |
| Male, no. (%) | 40 (51) |
| Age at diagnosis, y, median (range) | 37 (12-79) |
| Documented history of AA/MDS,* no. (%) | 24 (30) |
| Hemoglobinuria, no. (%) | 50 (63) |
| Abdominal pain, no. (%) | 24 (30) |
| Dysphagia, no. (%) | 9 (11) |
| Thrombosis, no. (%) | 4 (5) |
| Anemia (n = 73),† no. (%) | 69 (95) |
| LDH,‡ IU/L, median (range) | 2872 (587-10 300) |
| Thromboses before eculizumab (n) | |
| Venous | |
| Budd-Chiari | 12 |
| Mesenteric | 4 |
| Cerebral | 3 |
| Pulmonary embolus | 3 |
| Deep vein | 4 |
| Other | 3 |
| Arterial | |
| Stroke | 3 |
| Myocardial infarction | 2 |
| At start of eculizumab | |
| Age, y, median (range) | 46 (14-84) |
| LDH level, IU/L, median (range) | 2872 (587-10 300) |
| Neutrophils, ×109/L, median (range) | 2.2 (0.6-11.9) |
| Platelets, ×109/L, median (range) | 149 (11-507) |
| Reticulocytes (absolute), ×109/L, median (range) | 171 (57-415) |
| PNH clone sizes, % (range) | |
| Granulocyte | 96.4 (41.8-100) |
| Erythrocte | 34.0 (2.9-100) |
| Type II erythrocyte§ | 3.8 (0-77.4) |
| Type III erythrocyte§ | 25.0 (2.4-79.6) |
| Treatment at start of eculizumab, no. (%) | |
| Anticoagulation | 46 (58) |
| Ciclosporin | 11 (14) |
| Androgens | 2 (3) |
| No transfusion support‖ | 4 (5) |
One patient had myelodysplasia.
Hemoglobin at presentation was not known in 6 cases.
Normal value, 430 IU/L.
Type II erythrocytes have a partial deficiency, and type III erythrocytes have a complete deficiency of GPI-linked proteins.
Two patients refused to have blood transfusions for religious reasons.