Characteristics of the 3 subcategories of patients
| Characteristics . | Classic PNH . | Intermediate PNH . | AA-PNH . | P* . | P† . |
|---|---|---|---|---|---|
| No. of patients | 113 | 93 | 224 | ||
| Sex (%) | |||||
| Female | 61/113 (54) | 42/93 (45) | 132/224 (59) | .08 | .39 |
| Age at diagnosis, y, median | 40.6 | 31.9 | 34.5 | .006 | .002 |
| Diagnostic test | |||||
| Positive FC, no. (%) | 38/112 (34) | 52/93 (56) | 115/224 (51) | .002 | .003 |
| Size of PNH clone, median (n) | 46.0 (25) | 28.5 (42) | 20.0 (89) | <.001 | <.001 |
| GPI-negative cells more than 50% (%) | 12/25 (48) | 11/42 (26) | 13/89 (15) | .002 | <.001 |
| Presentation (%) | |||||
| Aplastic anemia preceding | 14/113 (12) | 29/93 (31) | 58/224 (26) | .003 | .004 |
| Signs and symptoms | |||||
| Clinical symptoms | |||||
| Abdominal pain | 37/113 (33) | 15/91 (17) | 27/224 (12) | <.001 | <.001 |
| Thrombosis | 13/113 (12) | 6/92 (7) | 11/222 (5) | .08 | .003 |
| Infections | 13/113 (12) | 9/92 (10) | 41/221 (19) | .07 | .10 |
| Peripheral blood abnormalities | |||||
| Anemia alone | 100/113 (89) | 1/93 (1) | 0 | <.001 | <.001 |
| Anemia and thrombocytopenia | 7/113 (6) | 40/93 (43) | 60/223 (27) | - | <.001 |
| Anemia and neutropenia | 0/113 | 7/93 (8) | 10/223 (5) | - | .02 |
| Pancytopenia at diagnosis | 0/113 | 13/93 (14) | 152/223 (68) | <.001 | <.001 |
| Initial treatment‡(%) | |||||
| No. of patients | 112 | 91 | 222 | ||
| None§ | 67/109 (61) | 55/91 (60) | 63/220 (29) | <.001 | <.001 |
| Drugs | |||||
| Danazol/androgens | 13/110 (12) | 22/91 (24) | 69/220 (31) | <.001 | <.001 |
| Corticosteroids alone | 17/109 (16) | 5/91 (5) | 13/221 (6) | .006 | .004 |
| Immunosuppressive treatment‖ | 4/110 (4) | 7/91 (8) | 73/222 (33) | <.001 | <.001 |
| Warfarin therapy | 12/110 (11) | 8/91 (9) | 6/222 (3) | .007 | .002 |
| Bone marrow transplantation | 0/112 (0) | 2/91 (2) | 23/222 (10) | <.001 | <.001 |
| Transfusions | 54/109 (50) | 25/90 (28) | 134/221 (61) | <.001 | .06 |
| Others | 12/110 (11) | 6/91 (7) | 19/219 (9) | .56 | .51 |
| Characteristics . | Classic PNH . | Intermediate PNH . | AA-PNH . | P* . | P† . |
|---|---|---|---|---|---|
| No. of patients | 113 | 93 | 224 | ||
| Sex (%) | |||||
| Female | 61/113 (54) | 42/93 (45) | 132/224 (59) | .08 | .39 |
| Age at diagnosis, y, median | 40.6 | 31.9 | 34.5 | .006 | .002 |
| Diagnostic test | |||||
| Positive FC, no. (%) | 38/112 (34) | 52/93 (56) | 115/224 (51) | .002 | .003 |
| Size of PNH clone, median (n) | 46.0 (25) | 28.5 (42) | 20.0 (89) | <.001 | <.001 |
| GPI-negative cells more than 50% (%) | 12/25 (48) | 11/42 (26) | 13/89 (15) | .002 | <.001 |
| Presentation (%) | |||||
| Aplastic anemia preceding | 14/113 (12) | 29/93 (31) | 58/224 (26) | .003 | .004 |
| Signs and symptoms | |||||
| Clinical symptoms | |||||
| Abdominal pain | 37/113 (33) | 15/91 (17) | 27/224 (12) | <.001 | <.001 |
| Thrombosis | 13/113 (12) | 6/92 (7) | 11/222 (5) | .08 | .003 |
| Infections | 13/113 (12) | 9/92 (10) | 41/221 (19) | .07 | .10 |
| Peripheral blood abnormalities | |||||
| Anemia alone | 100/113 (89) | 1/93 (1) | 0 | <.001 | <.001 |
| Anemia and thrombocytopenia | 7/113 (6) | 40/93 (43) | 60/223 (27) | - | <.001 |
| Anemia and neutropenia | 0/113 | 7/93 (8) | 10/223 (5) | - | .02 |
| Pancytopenia at diagnosis | 0/113 | 13/93 (14) | 152/223 (68) | <.001 | <.001 |
| Initial treatment‡(%) | |||||
| No. of patients | 112 | 91 | 222 | ||
| None§ | 67/109 (61) | 55/91 (60) | 63/220 (29) | <.001 | <.001 |
| Drugs | |||||
| Danazol/androgens | 13/110 (12) | 22/91 (24) | 69/220 (31) | <.001 | <.001 |
| Corticosteroids alone | 17/109 (16) | 5/91 (5) | 13/221 (6) | .006 | .004 |
| Immunosuppressive treatment‖ | 4/110 (4) | 7/91 (8) | 73/222 (33) | <.001 | <.001 |
| Warfarin therapy | 12/110 (11) | 8/91 (9) | 6/222 (3) | .007 | .002 |
| Bone marrow transplantation | 0/112 (0) | 2/91 (2) | 23/222 (10) | <.001 | <.001 |
| Transfusions | 54/109 (50) | 25/90 (28) | 134/221 (61) | <.001 | .06 |
| Others | 12/110 (11) | 6/91 (7) | 19/219 (9) | .56 | .51 |
Between the 3 subcategories of patients.
Between classic PNH and AA PNH.
In the first year after diagnosis.
No drugs, no warfarin, and no bone marrow transplantation in the first year.
Antithymocyte globulin and/or cyclosporine. Patients with intermediate PNH presented with anemia (n=61) either with or without other cytopenia; however, 32 had no anemia: 2 had thrombosis at diagnosis and neutropenia (n=1) or thrombocytopenia (n=1), and the 30 remaining had neutropenia and/or thrombocytopenia without reaching the criteria for AA-PNH subcategory (n=15; 6 had isolated neutropenia, and 9 patients did not have cytopenia).