Assessment and prognostic value of the European LeukemiaNet criteria for clinicohematologic response, resistance, and intolerance to hydroxyurea in polycythemia vera

Assessment and prognostic value of the European LeukemiaNet criteria for clinicohematologic response, resistance, and intolerance to hydroxyurea in polycythemia vera
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DOI:
10.1182/blood-2011-10-387787
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发表时间:
2012-02-09
期刊:
影响因子:
20.3
通讯作者:
Besses, Carles
Besses, Carles
中科院分区:
医学1区
文献类型:
--
作者:
Alvarez-Larran, Alberto;Pereira, Arturo;Besses, Carles

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欧洲白血病网络(ELN)提出了真性红细胞增多症(PV)对羟基脲(HU)的反应标准和耐药及不耐受的定义。在261例接受HU治疗的PV患者(中位随访时间为7.2年)中评价了这些标准,中位随访时间为4.4年。分别在24%、66%和10%的患者中观察到完全缓解、部分缓解和无缓解。达到ELN反应(完全或部分)或红细胞压积反应并没有导致更好的生存率或更少的血栓形成和出血。相反,白细胞计数无反应与较高的死亡风险相关(HR,2.7; 95%置信区间[CI],1.3%-5.4%; P = 0.007),而血小板计数无反应则涉及较高的血栓形成和出血风险。分别有11%和13%的患者对HU耐药和不耐受。对HU耐药与较高的死亡风险相关(HR,5.6; 95%CI,2.7%-11.9%; P
Criteria of response and definition of resistance and intolerance to hydroxyurea (HU) in polycythemia vera (PV) were proposed by the European LeukemiaNet (ELN). Such criteria were evaluated in 261 PV patients (median follow-up, 7.2 years) treated with HU for a median of 4.4 years. Complete response, partial response, and no response were observed in 24%, 66%, and 10% of patients, respectively. Achieving ELN response (complete or partial) or hematocrit response did not result in better survival or less thrombosis and bleeding. On the contrary, having no response in leukocyte count was associated with higher risk of death (HR, 2.7; 95% confidence interval [CI], 1.3%-5.4%; P = .007), whereas lack of response in platelet count involved a higher risk of thrombosis and bleeding. Resistance and intolerance to HU was registered in 11% and 13% of patients, respectively. Resistance to HU was associated with higher risk of death (HR, 5.6; 95% CI, 2.7%-11.9%; P