Final Results From PROUD-PV, A Randomized Controlled Phase 3 Trial Comparing Ropeginterferon Alfa-2b to Hydroxyurea in Polycythemia Vera Patients

摘要

An extended release formulation of anagrelide was evaluated in patients with ET in ARETA (Anagrelide Retard vs. Placebo: Efficacy and Safety in "At-Risk" Patients With Essential Thrombocythaemia), a parallel-group, patient- and sponsor-blinded, placebo-controlled, randomized phase 3 trial (Abstract 476). Eligible patients had a diagnosis of ET based on World Health Organization 2008 criteria, low platelet count, known JAK2 status, and at least 1 specified risk criterion. The study randomly assigned 146 patients to treatment, and 112 patients completed the first year of treatment. The trial met its primary endpoint, ET-related cardiovascular event-free survival (HR, 0.356; 95% CI, 0.16-0.79; P= 0008). In the extended-release anagrelide arm, 11.7% of patients progressed to high-risk status vs 26.1% in the placebo group (HR, 0.3/6; 95% CI, 0.16-0.81; P=.0048). Platelet counts normalized in the majority of patients treated with extended release anagrelide after 2 weeks of treatment. The safety profile was consistent with that observed with conventional anagrelide formulations, and included headache (41.6% with anagrelide vs 15.9% with placebo).