Table 1:
Representative overview of published literature on clinical outcomes of patients with ET and PV
| MPN Subtype | Study population (n) | Follow up (time) | Survival (time) | Thrombosis (all) | Thrombosis (arterial) | Thrombosis (venous) | Bleeding | Transformation |
|---|---|---|---|---|---|---|---|---|
| ET118 | 891 | 0-27 years (median 6.2) | Median overall survival 14.7 years (similar to sex and age standardized European population) | 22% at 15 years | Fibrotic 9%, Leukemic 2% (at 15 years) | |||
| ET47 (high vascular risk) | 809 (404 HU plus aspirin, 405 anagrelide plus aspirin) | 12-72 months (median 39) | Death 27/404 (hydroxyurea plus aspirin) 31/405 (anagrelide plus aspirin) |
17/404 (hydroxyurea plus aspirin) 37/405 (anagrelide plus aspirin) |
14/404 (hydroxyurea plus aspirin) 3/405 (anagrelide plus aspirin) |
Serious bleeding 8/404 (hydroxyurea plus aspirin) 22/405 (anagrelide plus aspirin) |
Myelofibrosis 5/404 (hydroxyurea plus aspirin), 16/405 anagrelide plus aspirin) AML or myelodysplasia 6/404 (hydroxyurea plus aspirin), 4/405 (anagrelide plus aspirin) |
|
| ET1, 119 (lacking high risk features for vascular events) | 382 (final analysis included 176 aspirin alone and 192 aspirin plus hydroxycarbamide | 0-187 months (median 73 months) | Death 7/176 (aspirin alone), 10/182 (aspirin plus hydroxycarbamide) | 7/176 (aspirin alone) 5/182 (aspirin plus hydroxycarbamide) |
3/176 (aspirin alone) 4/182 (aspirin plus hydroxycarbamide) |
Serious bleeding 2/176 (aspirin alone)3/182 (aspirin plus hydroxycarbamide) | Myelofibrosis 5/176 aspirin alone, 1/182 hydroxycarbamide plus aspirin AML 2/176 aspirin alone, 3/182 hydroxycarbamide plus aspirin. | |
| PV120 | 1638 | Mean 2.7 years, STDEV 1.3 years (4393 person years) | 3.7 deaths/100 patients/year (45% cardiovascular mortality, 13% haematological transformation) | 5.5/100 patients/year | Major bleeding 0.8/100 patients/year, any bleeding 2.9/100 patients/year |
Haematologic transformation 1.3/100patients/year | ||
| PV62 | 518 (253 aspirin, 265 placebo) | 1-3 years (1478 person-years) | Death 9/253 aspirin, 18/265 placebo | 8 out of 253 aspirin, 21/265 placebo | 5/253 aspirin, 13/265 placebo | 17/253 aspirin, 41/265 aspirin | Major bleeding 3/253 aspirin, 2/265 placebo. Any bleeding 23/253 aspirin 14/265 placebo | |
| PV43 | 365 (182 low target haematocrit <0.45, 183 high target haematocrit 0.45 to 0.5) | 1.5-48.1 mos (median 31) | Death 3/182 low haematocrit, 6/183 high haematocrit | 8/182 low haematocrit, 20/183 high haematocrit | 4/182 low haematocrit, 14/183 high haematocrit | 1/182 low haematocrit, 6/183 high haematocrit | 2/182 low haematocrit, 5/183 high haematocrit | Myelofibrosis 6/182 low haematocrit, 2/183 high haematocrit. AML or myelodysplasia 2/182 low haematocrit, 1/183 high haematocrit |