The goal of therapy for patients with essential thrombocythemia (ET) and polycythemia vera (PV) is to reduce thrombotic events by normalizing blood counts. Hydroxyurea (HU) and interferon-α (IFN-α) are the most frequently used cytoreductive options for patients with ET and PV at high risk for vascular complications. Myeloproliferative Disorders Research Consortium 112 was an investigator-initiated, phase 3 trial comparing HU to pegylated IFN-α (PEG) in treatment-naïve, high-risk patients with ET/PV. The primary endpoint was complete response (CR) rate at 12 months. A total of 168 patients were treated for a median of 81.0 weeks. CR for HU was 37% and 35% for PEG (P = .80) at 12 months. At 24 to 36 months, CR was 20% to 17% for HU and 29% to 33% for PEG. PEG led to a greater reduction in JAK2V617F at 24 months, but histopathologic responses were more frequent with HU. Thrombotic events and disease progression were infrequent in both arms, whereas grade 3/4 adverse events were more frequent with PEG (46% vs 28%). At 12 months of treatment, there was no significant difference in CR rates between HU and PEG. This study indicates that PEG and HU are both effective treatments for PV and ET. With longer treatment, PEG was more effective in normalizing blood counts and reducing driver mutation burden, whereas HU produced more histopathologic responses. Despite these differences, both agents did not differ in limiting thrombotic events and disease progression in high-risk patients with ET/PV. This trial was registered at www.clinicaltrials.gov as #NCT01259856.

中文翻译：

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干扰素-α 与羟基脲治疗真性红细胞增多症和原发性血小板增多症的随机 3 期试验。


原发性血小板增多症 (ET) 和真性红细胞增多症 (PV) 患者的治疗目标是通过使血细胞计数正常化来减少血栓事件。对于血管并发症高风险的 ET 和 PV 患者，羟基脲 (HU) 和干扰素-α (IFN-α) 是最常用的细胞减灭术选择。骨髓增殖性疾病研究联盟 112 是一项由研究者发起的 3 期试验，在未经治疗的高危 ET/PV 患者中比较 HU 与聚乙二醇化 IFN-α (PEG)。主要终点是 12 个月时的完全缓解 (CR) 率。共有 168 名患者接受治疗，中位时间为 81.0 周。 12 个月时，HU 的 CR 为 37%，PEG 的 CR 为 35% (P = .80)。 24 至 36 个月时，HU 的 CR 为 20% 至 17%，PEG 的 CR 为 29% 至 33%。 24 个月时，PEG 导致 JAK2V617F 大幅减少，但 HU 的组织病理学反应更频繁。血栓事件和疾病进展在两组中均很少见，而 PEG 组中 3/4 级不良事件则更为常见（46% vs 28%）。治疗 12 个月时，HU 和 PEG 之间的 CR 率没有显着差异。这项研究表明 PEG 和 HU 都是治疗 PV 和 ET 的有效方法。随着治疗时间的延长，PEG 在使血细胞计数正常化和减少驱动突变负担方面更有效，而 HU 产生更多的组织病理学反应。尽管存在这些差异，两种药物在限制 ET/PV 高危患者的血栓事件和疾病进展方面没有差异。该试验在 www.clinicaltrials.gov 上注册为#NCT01259856。