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MPD-RC 112

Trial question
Is either hydroxyurea or interferon-alpha superior over the other in high-risk patients with essential thrombocythemia or polycythemia vera?
Study design
Multi-center
Open label
RCT
Population
Characteristics of study participants
42.0% female
58.0% male
N = 168
168 patients (70 female, 98 male).
Inclusion criteria: high-risk patients with essential thrombocythemia or polycythemia vera.
Key exclusion criteria: primary myelofibrosis; prior malignancy within the last 5 years; contraindications to pegylated interferon or hydroxyurea; life-threatening comorbidities; history of active substance or alcohol abuse within last year.
Interventions
N=86 hydroxyurea (at a dose of 500 mg BID).
N=82 pegylated interferon-alpha (subcutaneous injection at 45 mcg weekly and titrated in 45 mcg increments monthly to a maximum of 180 mcg weekly).
Primary outcome
Complete response at 12 months
37%
35%
37.0 %
27.8 %
18.5 %
9.3 %
0.0 %
Hydroxyurea
Pegylated interferon-alpha
No significant difference ↔
No significant difference in complete response at 12 months (37% vs. 35%; ARD 2, 95% CI -13 to 16).
Secondary outcomes
No significant difference in overall response at 12 months (70% vs. 78%; ARD 8, 95% CI -5 to 21).
No significant difference in complete response at 24 months (20% vs. 29%; ARD 9, 95% CI -9 to 26).
No significant difference in complete response at 36 months (17% vs. 33%; ARD 17, 95% CI -8 to 40).
Safety outcomes
No significant differences in anemia, thrombocytopenia.
Significant differences in ≥ 3 grade adverse events (28% vs. 46%), grade 1-2 depression (3% vs. 15%).
Conclusion
In high-risk patients with essential thrombocythemia or polycythemia vera, hydroxyurea was not superior to pegylated interferon-alpha with respect to complete response at 12 months.
Reference
John Mascarenhas, Heidi E Kosiorek, Josef T Prchal et al. A randomized phase 3 trial of interferon-α vs hydroxyurea in polycythemia vera and essential thrombocythemia. Blood. 2022 May 12;139(19):2931-2941.
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