A multicenter study to assess efficacy, safety, and tolerability of ropeginterferon alfa-2b-njft in patients with essential thrombocythemia in the US and Canada: EXCEED-ET trial

作者：Lucia Masárová, Brandi Reeves, Firas El Chaer, Lynda Foltz, Tsewang Tashi, Ghaith Abu‐Zeinah, Jennifer Lucas, Anna B. Halpern, Dawn Maze, Albert Qin, Hana Safah, Fengshuo Lan, Casey L. O’Connell, Swati Goel, Lindsay Rein, Bruno Fang, Joan How, Sunil Babu, Zhuoyan Li, Sonia Cerquozzi, Stephen T. Oh, Anthony M. Hunter, Nikolai A. Podoltsev, Pankit Vachhani, Abdulraheem Yacoub, Julia Cunningham, Christopher Hillis, Salman Otoukesh, Oleh Zagrijtschuk, Henry Castro, Prithviraj Bose · 发表于：Frontiers in Medicine · 年份：2025 · DOI：10.3389/fmed.2025.1548590 · 被引用次数：4 · 研究领域：Myeloproliferative Neoplasms: Diagnosis and Treatment、Kruppel-like factors research、Eosinophilic Disorders and Syndromes

No new drugs have been approved for essential thrombocythemia (ET) treatment since the anagrelide approval in 1997. Ropeginterferon alfa-2b-njft (ropeg) is approved for polycythemia vera, providing a rationale for its use in ET. Its current dosing schema requires dose up-titrations with 50 mcg every 2 weeks and takes approximately 20 weeks to reach a plateau. The goal of this study is to assess the efficacy and safety of ropeg in ET using a higher initial dose and accelerated titration (HDAC) regimen. This is a single-arm, multicenter study in the US and Canada. Patients with ET receive ropeg at 250 mcg on Day 0, 350 mcg at Week 2, and 500 mcg from Week 4 onward with flexibility of dose adjustment. The primary endpoint is: platelets ≤400×10 9 /L, white blood cells <10×10 9 /L, improvement or non-progression of spleen size or major symptoms, and absence of hemorrhagic or thrombotic events, at months 10 and 13. Secondary endpoints include molecular response, safety and tolerability. A total of 91 patients were enrolled with 77 (84.6%) patients in the US and 14 (15.4%) in Canada. The last patient was enrolled on March 28, 2024. JAK2 V617F was found in 52 (57.1%) patients while CALR and MPL mutations in 34 (37.4%) and 5 (5.5%), respectively. As of November 12, 2024, the discontinuation rate was 8.8%. The study results will be available in mid-2025. This study will provide efficacy, tolerability and safety, molecular response and quality of life data that will be critical i...