Parasuraman Shreekant, DiBonaventura Marco, Reith Kelly, Naim Ahmad, Concialdi Kristen, Sarlis Nicholas J
Incyte Corporation, 1801 Augustine Cut-Off, Wilmington, DE 19803 USA.
Kantar Health, 11 Madison Avenue, 12th Floor, New York, NY 10010 USA.
Exp Hematol Oncol. 2016 Feb 1;5:3. doi: 10.1186/s40164-016-0031-8. eCollection 2015.
Hydroxyurea (HU) is among the most commonly used cytoreductive treatments for polycythemia vera (PV), but previous research and clinical experience suggest that not all patients respond optimally, consistently, or durably to HU treatment. This study investigated patterns of HU use and impact on disease control among patients with PV in real-world clinical practice in the United States.
Oncologists and hematologists recruited between April and July 2014 reported data from patient charts. Treatment history and disease symptom comparisons between HU subgroups were performed using Chi square tests or one-way analyses of variance for categorical and continuous variables. Other analyses were performed using descriptive statistics.
Overall, 329 physicians participated and provided data on 1309 patients with PV (62.3 % male; mean age = 62.5 years, mean time since diagnosis = 5.2 years). In the 229 (17.5 %) patients who had stopped HU, the most common reasons for HU discontinuation-as assessed by the treating clinician-were inadequate response (29.3 %), intolerance (27.5 %), and disease progression (12.7 %). Among patients currently on HU, a significant proportion had elevated blood cell counts: 34.4 % had hematocrit values ≥45 %, 59.4 % had platelet levels >400 × 10(9)/L, and 58.2 % had WBC counts > 10 × 10(9)/L. Two-thirds (66.3 %) of patients had ≥1 elevated count, 40.3 % had ≥2 elevated counts, and 19.8 % had all 3 counts elevated. The most common PV-related signs and symptoms among all patients were fatigue and splenomegaly.
Although many patients with PV benefit from HU therapy, some continue to have suboptimal control of their disease, as evidenced by persistence of abnormally elevated blood cell counts and the continued experience of disease-related manifestations (signs and symptoms). These data further denote a significant medical need for some patients with PV currently or previously treated with HU.
羟基脲（HU）是真性红细胞增多症（PV）最常用的细胞减灭治疗药物之一，但既往研究和临床经验表明，并非所有患者对HU治疗都能达到最佳、持续或持久的反应。本研究调查了美国真实世界临床实践中PV患者使用HU的模式及其对疾病控制的影响。
2014年4月至7月间招募的肿瘤学家和血液学家报告了患者病历数据。使用卡方检验或对分类变量和连续变量进行单向方差分析，对HU亚组之间的治疗史和疾病症状进行比较。其他分析采用描述性统计方法。
总体而言，329名医生参与并提供了1309例PV患者的数据（男性占62.3%；平均年龄=62.5岁，平均诊断后时间=5.2年）。在229例（17.5%）停用HU的患者中，经治疗医生评估，停用HU最常见的原因是反应不足（29.3%）、不耐受（27.5%）和疾病进展（12.7%）。在目前正在使用HU的患者中，很大一部分患者血细胞计数升高：34.4%的患者血细胞比容值≥45%，59.4%的患者血小板水平>400×10⁹/L，58.2%的患者白细胞计数>10×10⁹/L。三分之二（66.3%）的患者有≥1项计数升高，40.3%的患者有≥2项计数升高，19.8%的患者三项计数均升高。所有患者中最常见的PV相关体征和症状是疲劳和脾肿大。
尽管许多PV患者从HU治疗中获益，但仍有一些患者疾病控制不佳，表现为血细胞计数持续异常升高以及持续出现与疾病相关的表现（体征和症状）。这些数据进一步表明，目前或既往接受HU治疗的一些PV患者存在重大医疗需求。
