研究动态
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羟基脲不耐受对真性红细胞增多症在常规临床实践中的临床和经济影响。

Clinical and Economic Implications of Hydroxyurea Intolerance in Polycythemia Vera in Routine Clinical Practice.

发表日期:2024 Jun 10
作者: Martin H Ellis, Tamar Tadmor, Naama Yekutiel, Gabriel Chodick, Moti Levy, Giora Sharf, Nana Ben Zvi, Raanan Leef, Oren Feine, Oren Shavit
来源: Journal of Hematology & Oncology

摘要:

背景/目标:真性红细胞增多症(PV)是一种慢性血液肿瘤,通常用羟基脲(HU)治疗。我们利用 Maccabi Healthcare Services 的先进数字化数据库回顾性研究了 HU 不耐受在以色列 PV 患者常规临床护理中的临床和经济影响。方法:我们收集了有关人口统计、就诊、住院、实验室结果、药物购买、心血管和血栓事件、心理健康、经济结果和死亡率的数据。结果包括心血管和其他血栓事件、疾病进展、心理健康事件、经济结果和总体死亡率。结果:在研究的830名患者中,3名(0.4%)对HU治疗耐药,318名(38.3%)对HU治疗不耐受,509名(61.3%)对HU治疗稳定。与 HU 稳定的患者相比,HU 不耐受患者的静脉血栓形成率显着更高(分别为每 100 人年 1.58 和 0.47 [PY];p < 0.001)。 HU 不耐受患者与 HU 稳定患者中,每 100 PY 进展为骨髓纤维化的比率分别为 6 例和 0.9 例(p < 0.001),进展为急性髓系白血病 (AML) 的比率分别为 1.16 例和 0.2 例HU 不耐受患者与 HU 稳定患者的每 100 PY 分别为 (p < 0.001)。 HU 不耐受患者的放血需求、死亡率和总住院天数显着高于 HU 稳定患者(分别为 p = 0.049、p < 0.001、p < 0.001)。与 HU 稳定患者相比,HU 不耐受患者发生更多心理健康相关事件 (p = 0.007),与 HU 稳定患者相比,HU 不耐受患者的总医疗费用比率为 2.65。结论:本研究表明,HU 不耐受的患者比 HU 稳定的患者更有可能出现更差的结果,强调需要密切监测这些患者的疾病相关并发症或进展。
Background/Objectives: Polycythemia vera (PV) is a chronic hematologic neoplasm commonly treated with hydroxyurea (HU). We utilized the advanced digitalized database of Maccabi Healthcare Services to retrospectively investigate the clinical and economic implications of HU intolerance in the routine clinical care of PV patients in Israel. Methods: We collected data on demographics, physician visits, hospitalizations, laboratory results, medication purchases, cardiovascular and thrombotic events, mental health, economic outcomes, and mortality. Outcomes included cardiovascular and other thrombotic events, disease progression, mental health events, economic outcomes, and overall mortality. Results: Of the 830 patients studied, 3 (0.4%) were resistant to HU treatment, 318 (38.3%) were intolerant to HU treatment, and 509 (61.3%) were stable on HU treatment. The venous thrombosis rate was significantly higher among HU-intolerant compared to HU-stable patients (1.58 vs. 0.47 per 100 person-years [PY], respectively; p < 0.001). The rate of progression to myelofibrosis was 6 vs. 0.9 per 100 PY in HU-intolerant patients vs. HU-stable patients, respectively (p < 0.001), and the rate of progression to acute myeloid leukemia (AML) was 1.16 vs. 0.2 per 100 PY in HU-intolerant patients vs. HU-stable patients, respectively (p < 0.001). The phlebotomy requirement, mortality rate, and total hospitalization days among HU-intolerant patients were significantly higher than in HU-stable patients (p = 0.049, p < 0.001, p < 0.001, respectively). More mental health-related events were noted in HU-intolerant patients vs. HU-stable patients (p = 0.007), and the total healthcare cost ratio was 2.65 for the HU-intolerant patients compared with HU-stable patients. Conclusions: This study suggests that HU-intolerant patients are more likely to have worse outcomes than HU-stable patients, highlighting the need for the close monitoring of these patients for disease-related complications or progression.