Ropeginterferon alfa-2b versus anagrelide for the treatment of essential thrombocythemia: Topline results of the phase 3 SURPASS-ET trial.

作者：Ruben A. Mesa, Harinder Gill, Zhijian Xiao, Norio Komatsu, Albert Qin, Tsewang Tashi, Lei Zhang, Jie Jin, Keita Kirito, Kohshi Ohishi, Shuichi Shirane, Lee-Yung Shih, Sung Eun Lee, Winnie Z.Y. Teo, Dawn Maze, Stephen T. Oh, Toshiaki Sato, Weichung Joe Shih, John Mascarenhas, Lucia Masárová, SURPASS-ET Study Group · 发表于：Journal of Clinical Oncology · 年份：2025 · DOI：10.1200/jco.2025.43.16_suppl.6500 · 被引用次数：4 · 研究领域：Myeloproliferative Neoplasms: Diagnosis and Treatment、Chronic Myeloid Leukemia Treatments、Kruppel-like factors research

6500 Background: BCR::ABL1 -negative myeloproliferative neoplasms (MPNs), including essential thrombocythemia (ET), polycythemia vera (PV) and myelofibrosis (MF). No new treatments have been approved for ET in the US since anagrelide in 1997. Ropeginterferon alfa-2b (ropeg) is an anti-clonal interferon-based therapy approved by the FDA and globally for PV, providing the rationale for evaluation in ET and other MPNs. Methods: SURPASS ET is an open-label, multicenter, Phase III trial comparing ropeg with anagrelide over 12 months in patients with ET who were hydroxyurea-resistant or-intolerant. A total of 174 patients were randomized in a 1:1 ratio to receive ropeg or anagrelide. Ropeg was administered at 250 mcg at Week 0 titrating to 350 mcg at Week 2 and 500 mcg from Week 4 if tolerated. The primary endpoint was the rate of response, as defined by the Modified European LeukemiaNet response criteria, at both Months 9 and 12. Secondary endpoint assessments included JAK2 V617F allele burden, MPN-associated symptoms, thromboembolic events, spleen size, and safety. Results: Baseline patient characteristics were balanced across treatment arms (Table 1). The primary endpoint was achieved in 42.9% of patients in the ropeg arm versus 6.0% in the anagrelide arm (p=0.0001). Ropeg showed response improvements by each parameter: 1) Platelets ≤400x10 9 /L and white blood cells <9.5x10 9 /L: 56.0% vs. 6.0%. 2) Improvement or stabilization of splenomegaly: 87.9% vs. 54.2%. 3) Symptom imp...