To compare the efficacy and safety of gecacitinib (also known as jaktinib) with hydroxyurea (HU) in treating myelofibrosis (MF) patients. In this multicenter, randomized phase 3 trial (ZGJAK016), intermediate- or high-risk primarily JAK inhibitor naïve MF patients were assigned in a 2:1 ratio to receive either gecacitinib (100 mg twice a day, BID) or HU (500 mg BID). The primary endpoint was the proportion of patients with ≥35% reduction in spleen volume (SVR35) from baseline at week 24. Secondary endpoints included the best spleen response rate, the proportion of patients with a ≥50% reduction in total symptom score (TSS50), anemia improvement, and safety profile. At 24 weeks, the SVR35 was reached by 64.8% of patients on gecacitinib (46/71), compared to 26.5% on HU (9/34), P = 0.0002. The best spleen response rates were also superior for gecacitinib at 81.7%, vs 32.4% for HU, P < 0.0001. The TSS50 rates were 62.0% for gecacitinib- and 50% for HU-treated patients. Among non-transfusion-dependent patients with baseline hemoglobin (HGB) ≤ 100 g/L, 31.0% (13/42) in the gecacitinib group showed a ≥20 g/L increase in HGB, compared to 15.0% (3/20) in HU group. The common grade ≥ 3 treatment-emergent adverse events (TEAEs), including anemia (26.8% vs 44.1%), thrombocytopenia (15.5% vs 32.4%), leukopenia (2.8% vs 20.6%), and neutropenia (1.4% vs 20.6%), were less frequent with gecacitinib than HU. Treatment discontinuation due to TEAEs was lower in gecacitinib (7.0%) compared to HU (11.8%). Gecacitinib demonstrates superior efficacy and a more favorable safety profile compared to HU, making it a promising treatment option for managing MF, particularly in patients with anemia (This trial was registered with ClinicalTrials.gov, (NCT04617028)).
比较吉卡替尼（亦称为贾克替尼）与羟基脲（HU）治疗骨髓纤维化（MF）患者的疗效和安全性。在这项多中心、随机、3期试验（ZGJAK016）中，中危或高危、主要未使用过JAK抑制剂的MF患者按2:1比例分配接受吉卡替尼（100毫克，每天两次）或HU（500毫克，每天两次）。主要终点为第24周时脾脏体积从基线减少≥35%（SVR35）的患者比例。次要终点包括最佳脾脏反应率、总症状评分减少≥50%（TSS50）的患者比例、贫血改善和安全性。第24周时，吉卡替尼组的SVR35达到64.8%（46/71），而HU组为26.5%（9/34），P=0.0002。吉卡替尼的最佳脾脏反应率也更高，为81.7%，而HU组为32.4%，P<0.0001。吉卡替尼组的TSS50率为62.0%，HU治疗患者为50%。在非输血依赖且基线血红蛋白（HGB）≤100 g/L的患者中，吉卡替尼组31.0%（13/42）显示血红蛋白增加≥20 g/L，而HU组为15.0%（3/20）。常见≥3级治疗期间出现的不良事件（TEAEs），包括贫血（26.8% vs 44.1%）、血小板减少（15.5% vs 32.4%）、白细胞减少（2.8% vs 20.6%）和中性粒细胞减少（1.4% vs 20.6%），吉卡替尼组发生率低于HU组。因TEAEs停药的比例，吉卡替尼组（7.0%）低于HU组（11.8%）。吉卡替尼显示出优于HU的疗效和更良好的安全性，使其成为治疗MF的有前景的选择，特别是对于贫血患者（本试验已在ClinicalTrials.gov注册，编号NCT04617028）。
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