Faculty of Medicine, Department of Internal Medicine, Chiang Mai University, Chiang Mai, Thailand.
Hematology. 2022 Dec;27(1):813-819. doi: 10.1080/16078454.2022.2105582.
To determine the prevalence, clinical outcomes, and factors associated with hydroxyurea (HU) resistance or intolerance among polycythemia vera (PV) and essential thrombocythemia (ET) patients.
This study was a retrospective cohort study including PV and ET patients diagnosed by WHO criteria and treated with HU between January 2000 and June 2020. Clinical features, laboratory data, and resistance or intolerance of HU were collected. The prevalence, clinical outcomes, and associated factors of HU resistance or intolerance were analyzed.
There were 260 patients including 144 ET and 116 PV. The prevalence of HU resistance or intolerance was 11.9% (31 patients) which was more frequent in ET patients (14.6% vs. 8.6% in PV). Patients who had HU resistance or intolerance significantly increased the risk of bleeding events (HR 2.64; 95% CI 1.19-5.85, = 0.017). The risk factors of HU resistance or intolerance were low baseline hemoglobin levels (HR 0.90; 95%CI 0.84-0.97, = 0.01), age more than 60 years old (HR 3.98; 95% CI 2.08-7.62, < 0.001) and splenomegaly (HR 2.08; 95% CI 1.03-4.21, = 0.04).
The prevalence of HU resistance or intolerance in PV and ET patients was 11.9%. Patients with HU resistance or intolerance significantly increased the risk of bleeding complications.
目的 确定真性红细胞增多症（PV）和原发性血小板增多症（ET）患者使用羟基脲（HU）治疗过程中出现耐药或不耐受的发生率、临床结局及相关因素。
本研究为回顾性队列研究，纳入 2000 年 1 月至 2020 年 6 月期间经世界卫生组织（WHO）标准诊断为 PV 和 ET 并接受 HU 治疗的患者。收集临床特征、实验室数据以及 HU 耐药或不耐受情况。分析 HU 耐药或不耐受的发生率、临床结局及相关因素。
共纳入 260 例患者，其中 ET 患者 144 例，PV 患者 116 例。HU 耐药或不耐受的发生率为 11.9%（31 例），ET 患者的发生率（14.6%）高于 PV 患者（8.6%）。HU 耐药或不耐受患者发生出血事件的风险显著增加（HR 2.64；95%CI 1.19-5.85， = 0.017）。HU 耐药或不耐受的危险因素包括基线血红蛋白水平较低（HR 0.90；95%CI 0.84-0.97， = 0.01）、年龄大于 60 岁（HR 3.98；95%CI 2.08-7.62， < 0.001）和脾肿大（HR 2.08；95%CI 1.03-4.21， = 0.04）。
PV 和 ET 患者 HU 耐药或不耐受的发生率为 11.9%。HU 耐药或不耐受患者发生出血并发症的风险显著增加。
