Yacoub Abdulraheem, Lyons Roger, Verstovsek Srdan, Shao Ryan, Chu David Tin, Agrawal Apurv, Sivaraman Smitha, Colucci Philomena, Paranagama Dilan, Mascarenhas John
The Division of Hematologic Malignancies and Cellular Therapeutics, University of Kansas Medical Center, Westwood, KS.
Texas Oncology and US Oncology Research, San Antonio, TX.
Clin Lymphoma Myeloma Leuk. 2021 Jul;21(7):461-469. doi: 10.1016/j.clml.2021.02.011. Epub 2021 Mar 1.
Few data exist regarding the disease and clinical characteristics of patients with essential thrombocythemia (ET) in the United States. The ongoing, multicenter, noninterventional, prospective, Myelofibrosis and Essential Thrombocythemia Observational STudy (MOST) was designed to collect data pertaining to the demographics, clinical management, and patient-reported outcomes in patients with myelofibrosis or ET in the United States (NCT02953704). This analysis examines the clinical characteristics of patients with clinical diagnoses of high-risk or low-risk ET receiving ET-directed therapy at enrollment. At data cutoff (June 17, 2019), 1207 of 1234 enrolled patients were eligible for this analysis (median age, 70 years; 65% female; 88% white); 917 patients (76%) had mutation testing results available. The median time from ET diagnosis to study enrollment was 4.2 years. The majority of patients (87%) had high-risk ET. Of 333 patients with a history of thrombotic events, 247 had at least 1 event classified as arterial and/or venous. Platelet count was above normal range in 54% of patients. Hypertension (56%) was the most common comorbidity. At enrollment, the majority of patients (low-risk ET, 94%; high-risk ET, 79%) were receiving ET-directed monotherapy. Additional prospective analyses from MOST will help to identify areas of unmet need.
在美国，关于原发性血小板增多症（ET）患者的疾病及临床特征的数据很少。正在进行的多中心、非干预性、前瞻性的骨髓纤维化和原发性血小板增多症观察性研究（MOST）旨在收集美国骨髓纤维化或ET患者的人口统计学、临床管理及患者报告结局相关的数据（NCT02953704）。本分析研究了入组时接受ET定向治疗的高危或低危ET临床诊断患者的临床特征。在数据截止时（2019年6月17日），1234名入组患者中有1207名符合本分析条件（中位年龄70岁；65%为女性；88%为白人）；917名患者（76%）有可用的突变检测结果。从ET诊断到研究入组的中位时间为4.2年。大多数患者（87%）为高危ET。在333名有血栓形成事件史的患者中，247名至少有1次事件分类为动脉和/或静脉血栓形成。54%的患者血小板计数高于正常范围。高血压（56%）是最常见的合并症。入组时，大多数患者（低危ET患者为94%；高危ET患者为79%）正在接受ET定向单药治疗。MOST的其他前瞻性分析将有助于确定未满足需求的领域。
