CYTO-PV
Trial question
What is the role of hematocrit target < 45% in patients with polycythemia vera?
Study design
Multi-center
Open label
RCT
Population
Characteristics of study participants
38.0% female
62.0% male
N = 365
365 patients (138 female, 227 male)
Inclusion criteria: adult patients with JAK2-positive polycythemia vera who were being treated with phlebotomy, hydroxyurea, or both
Key exclusion criteria: hypersensitivity or contraindication to study treatments, significant liver or renal disease, history of active substance or alcohol abuse within the last year, pregnant or lactating women, presence of any life-threatening condition or of any disease that is likely to significantly shorten life expectancy
Interventions
N=182 low-hematocrit targeted therapy (more aggressive therapy for a hematocrit target of < 45%)
N=183 high-hematocrit targeted therapy (less aggressive therapy for a hematocrit target of 45-50%)
Primary outcome
Death from cardiovascular causes or major thrombotic events
2.7
9.8
9.8 %
7.4 %
4.9 %
2.5 %
0.0 %
Low-hematocrit targeted
therapy
High-hematocrit targeted
therapy
Significant
decrease ▼
NNT = 14
Significant decrease in death from cardiovascular causes or major thrombotic events (2.7% vs. 9.8%; HR 0.26, 95% CI 0.1 to 0.69)
Secondary outcomes
Significant decrease in total cardiovascular events (4.4% vs. 10.9%; HR 0.37, 95% CI 0.16 to 0.84)
Safety outcomes
No significant difference in rate of adverse events.
Conclusion
In adult patients with JAK2-positive polycythemia vera who were being treated with phlebotomy, hydroxyurea, or both, low-hematocrit targeted therapy was superior to high-hematocrit targeted therapy with respect to death from cardiovascular causes or major thrombotic events.
Reference
Marchioli R, Finazzi G, Specchia G et al. Cardiovascular events and intensity of treatment in polycythemia vera. N Engl J Med. 2013 Jan 3;368(1):22-33.
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