Clinical, biologic, and initial treatment of 460 patients with PNH
| Characteristic . | n/N (%) or median (IQR*), N . |
|---|---|
| Sex, female | 250/460 (54.3%) |
| Age at diagnosis, y | 34.2 (24.2-48.4)#, 460 |
| Diagnostic test | |
| Flow cytometry† | 215/459 (46.8%) |
| Size of PNH clone (%) | 30 (14-50), 159 |
| GPI-negative cells more than 50% | 37/159 (23%) |
| Presentation | |
| Aplastic anemia preceding | 106/460 (23.0%) |
| Signs and symptoms | |
| Clinical symptoms | |
| Abdominal pain | 83/456 (18.2%) |
| Thrombosis‡ | 33/457 (7.2%) |
| Infections | 69/453 (15.2%) |
| Peripheral blood abnormalities | |
| Anemia alone | 102/447 (22.8%) |
| Anemia and thrombocytopenia | 113/447 (25.3%) |
| Anemia and neutropenia | 18/447 (4.0%) |
| Pancytopenia at diagnosis | 175/447 (39.1%) |
| Initial treatment§ | 454 |
| None‖ | 193/448 (43.1%) |
| Drug | |
| Danazole/androgens | 110/449 (24.5%) |
| Corticosteroids alone | 40/449 (8.9%) |
| Immunosuppressive treatment¶ | 92/451 (20.4%) |
| Warfarin therapy | 29/451 (6.4%) |
| Bone marrow transplantation | 26/454 (5.7%) |
| Transfusions | 226/447 (50.6%) |
| Others | 40/448 (8.9%) |
| Characteristic . | n/N (%) or median (IQR*), N . |
|---|---|
| Sex, female | 250/460 (54.3%) |
| Age at diagnosis, y | 34.2 (24.2-48.4)#, 460 |
| Diagnostic test | |
| Flow cytometry† | 215/459 (46.8%) |
| Size of PNH clone (%) | 30 (14-50), 159 |
| GPI-negative cells more than 50% | 37/159 (23%) |
| Presentation | |
| Aplastic anemia preceding | 106/460 (23.0%) |
| Signs and symptoms | |
| Clinical symptoms | |
| Abdominal pain | 83/456 (18.2%) |
| Thrombosis‡ | 33/457 (7.2%) |
| Infections | 69/453 (15.2%) |
| Peripheral blood abnormalities | |
| Anemia alone | 102/447 (22.8%) |
| Anemia and thrombocytopenia | 113/447 (25.3%) |
| Anemia and neutropenia | 18/447 (4.0%) |
| Pancytopenia at diagnosis | 175/447 (39.1%) |
| Initial treatment§ | 454 |
| None‖ | 193/448 (43.1%) |
| Drug | |
| Danazole/androgens | 110/449 (24.5%) |
| Corticosteroids alone | 40/449 (8.9%) |
| Immunosuppressive treatment¶ | 92/451 (20.4%) |
| Warfarin therapy | 29/451 (6.4%) |
| Bone marrow transplantation | 26/454 (5.7%) |
| Transfusions | 226/447 (50.6%) |
| Others | 40/448 (8.9%) |
Interquartile range.
Flow cytometry clone size was determined at diagnosis from granulocytes. It was available among 159 of 215 patients diagnosed by flow cytometry.
Location of thrombosis at diagnosis includes 16 Budd Chiari.
In the first year after diagnosis, data were available for 454 patients.
No drugs, no warfarin, and no bone marrow transplantation.
Antithymocyte globulin and/or cyclosporine.
Sixty-six (14%) patients in our study were between 6 and 20 years old and 15 patients were younger than 16 years old at diagnosis.