Clinical course and complications in the global population, N = 454
| Complications . | n/N (%) . | 10-year cumulative incidence rate, % (95% CI) . |
|---|---|---|
| Bicytopenia or pancytopenia | 36/231 (at risk)§ | 19.2 (12.9-25.2) |
| Recurrent abdominal pain crisis | 138/451 (30.2) | NA |
| Infections* | 185/453 (40.8) | NA |
| Thrombosis | 116/454‖ | 30.7 (25.4-35.9)¶ |
| Budd-Chiari syndrome | 49 (43) | |
| Central nervous system | 35 (31) | |
| Limbs | 31 (27) | |
| Other sites | 29 (25) | |
| Myelodysplastic syndrome† | 21/454 | 5.2 (2.9%-7.6%) |
| Acute leukemia† | 8/454 | 2.4 (0.7%-4.0%) |
| Deaths‡ | 96/454 | |
| Central nervous system (SCN) vascular complications | 23 | NA |
| Infectious diseases | 23 | NA |
| Budd-Chiari syndrome | 21 | NA |
| Malignant disorders | 9 | NA |
| Other causes | 17 | NA |
| Complications . | n/N (%) . | 10-year cumulative incidence rate, % (95% CI) . |
|---|---|---|
| Bicytopenia or pancytopenia | 36/231 (at risk)§ | 19.2 (12.9-25.2) |
| Recurrent abdominal pain crisis | 138/451 (30.2) | NA |
| Infections* | 185/453 (40.8) | NA |
| Thrombosis | 116/454‖ | 30.7 (25.4-35.9)¶ |
| Budd-Chiari syndrome | 49 (43) | |
| Central nervous system | 35 (31) | |
| Limbs | 31 (27) | |
| Other sites | 29 (25) | |
| Myelodysplastic syndrome† | 21/454 | 5.2 (2.9%-7.6%) |
| Acute leukemia† | 8/454 | 2.4 (0.7%-4.0%) |
| Deaths‡ | 96/454 | |
| Central nervous system (SCN) vascular complications | 23 | NA |
| Infectious diseases | 23 | NA |
| Budd-Chiari syndrome | 21 | NA |
| Malignant disorders | 9 | NA |
| Other causes | 17 | NA |
NA indicates not applicable.
At least 1 severe microbiologically proven bacterial infection.
Four patients developed both myelodysplastic syndrome and acute leukemia; the 10-year cumulative incidence of both MDS and AL is 3.8% (95% CI; 0.1-7.5). The median time (interquartile range) from diagnosis to myelodysplasia and acute leukemia was 1.3 (0.5-4.8) and 1.6 (0.8-4.4) years, respectively.
Three unknown causes of death; main causes of death included 9 Budd-Chiari syndrome, 6 CNS vascular complications, and 8 infections for classical PNH (n = 113); 5 Budd-Chiari syndrome, 11 CNS vascular complications, and 8 infections for AA-PNH syndrome (n = 222); and 2 Budd-Chiari syndrome, 5 CNS vascular complications, and 4 infections for unclassified PNH (n = 91).
Patients with pancytopenia at diagnosis were excluded from the group of patients at risk. Cumulative incidence of bicytopenia or pancytopenia was thus calculated for 231 patients.
Location of thrombosis unknown for 2 patients.
Cumulative incidence of thrombosis was calculated for 114 patients (unknown data for 2 patients). The median time (interquartile range) from diagnosis to bicytopenia or tricytopenia and first thrombosis was 2.1 (0.9-5.4) and 2.3 (1.1-6.5) years, respectively.