Department of Hematologic Oncology and Blood Disorders, Levine Cancer Institute, Atrium Health, Charlotte, NC.
US Oncology Hematology Research Program, Rocky Mountain Cancer Centers, Aurora, CO.
Clin Lymphoma Myeloma Leuk. 2019 Sep;19(9):579-584.e1. doi: 10.1016/j.clml.2019.06.001. Epub 2019 Jun 13.
Approximately 50% of patients with polycythemia vera (PV) have PV-related symptoms at diagnosis; these symptoms might develop or worsen with time. Symptoms have been shown to negatively affect quality of life and interfere with daily activities. To our knowledge, an analysis to evaluate the relationship between blood count control and symptoms has not been published.
The Prospective Observational Study of Patients with Polycythemia Vera in US Clinical Practices (REVEAL; NCT02252159) is a multicenter, noninterventional, nonrandomized prospective observational study of patients with PV in the United States. Patients included were required to have a complete blood count result within 30 days before completing the at-enrollment Myeloproliferative Neoplasm Self-Assessment Form Total Symptom Score (MPN-SAF TSS). Symptom severity was compared between those who had blood count control versus those who did not.
At the time of enrollment, 1714 patients (94.5%) were being managed with cytoreductive therapy; 468 patients (25.8%) had complete hematologic remission (CHR), 1614 patients (89.0%) had ≥1 controlled blood count, and 1122 patients (61.9%) had ≥2 controlled blood counts. Mean MPN-SAF TSSs were similar across patients in different blood count control groups. Fatigue was the most frequently reported symptom. The severity of individual symptoms, except those of pruritus and night sweats, was not affected by CHR or the number of blood counts that were controlled.
Symptom burden in patients with PV can persist despite control of blood counts, which suggests some discordance between laboratory values and symptom burden. Consequently, regular monitoring of symptom burden should be factored into the assessment of disease control.
约 50% 的真性红细胞增多症 (PV) 患者在诊断时存在与 PV 相关的症状;这些症状可能会随时间发展或恶化。症状已被证明会对生活质量产生负面影响,并干扰日常活动。据我们所知,目前尚未发表分析血液计数控制与症状之间关系的研究。
美国真性红细胞增多症临床实践的前瞻性观察研究 (REVEAL;NCT02252159) 是一项在美国进行的多中心、非干预性、非随机的前瞻性观察性研究,纳入了真性红细胞增多症患者。要求患者在完成入组时的骨髓增殖性肿瘤自我评估表总症状评分 (MPN-SAF TSS) 前 30 天内有一次完整的全血细胞计数结果。比较了有血液计数控制的患者与无血液计数控制的患者之间的症状严重程度。
在入组时,1714 例患者(94.5%)正在接受细胞减少治疗;468 例患者(25.8%)达到完全血液学缓解(CHR),1614 例患者(89.0%)有≥1 次受控的血液计数,1122 例患者(61.9%)有≥2 次受控的血液计数。不同血液计数控制组的患者 MPN-SAF TSS 均值相似。疲劳是最常报告的症状。除瘙痒和盗汗外,个别症状的严重程度不受 CHR 或受控血液计数数量的影响。
尽管控制了血液计数,但 PV 患者的症状负担仍然存在,这表明实验室值与症状负担之间存在一些不一致。因此,应将症状负担的定期监测纳入疾病控制的评估中。