背景:真性红细胞增多症(PV)和原发性血小板增多症(ET)患者的治疗是根据明确定义的风险分层系统进行的。我们假设遵守准则,即决定避免在非高危患者中引入细胞减灭术,对于年龄在40至59岁之间的患者(中间年龄组)尤其困难。
目的:为了评估中年PV和ET患者的组,专注于诊断时适应的一线治疗方法。
方法:研究组由从6个波兰成人白血病组(PALG)中心招募的308名PV和ET患者组成。对患者进行疾病表型分析,风险组,治疗方法,心血管(CV)危险因素,出血或血栓形成的发生。
结果:总体而言,研究组中74%的患者在诊断时开始进行细胞减灭术,包括70%的低危PV患者和85-89%的非高危ET患者。影响决定开始治疗的因素包括较高的血红蛋白(Hb)浓度(PV)以及较高的血小板(PLT)计数,和CV危险因素的存在(ET)。在诊断时引入细胞减灭术对血栓事件没有影响。有CV危险因素的患者在诊断和随访时都经历了较高的并发症发生率。独立于治疗策略。
结论:我们强调了中年PV和ET患者对建议的依从性低。此外,我们强调CV危险因素的重要性,并强调其对该患者人群疾病表型的影响.
BACKGROUND: The treatment of patients with polycythemia vera (PV) and essential thrombocythemia (ET) is conducted according to well-defined risk stratification systems. We hypothesized that adherence to the guidelines, namely the decision to refrain from introducing
cytoreduction in non-high-risk patients, is particularly difficult in patients diagnosed when they are between 40 and 59 years of age (intermediate-age group).
OBJECTIVE: To evaluate the group of intermediate-age PV and ET patients, focusing on a first-line treatment approach adapted at diagnosis.
METHODS: The study group consisted of 308 PV and ET patients recruited from 6 Polish Adult Leukemia Group (PALG) Centers. Patients were analyzed with respect to disease phenotype, risk group, treatment approach, cardiovascular (CV) risk factors, and occurrence of bleeding or thrombosis.
RESULTS: Overall, 74% of patients in the study group were started on
cytoreduction at diagnosis, including 70% of the low-risk PV patients and 85-89% of the non-high-risk ET patients. Factors influencing the decision to start the treatment included higher hemoglobin (Hb) concentration (in PV) as well as higher platelet (PLT) count, and the presence of CV risk factors (in ET). Introducing
cytoreduction at diagnosis had no impact on thrombotic events. Patients harboring CV risk factors experienced a higher incidence of complications both at diagnosis and follow-up, independently of the treatment strategy.
CONCLUSIONS: We underline the low adherence to recommendations in the treatment of intermediate-age PV and ET patients. Moreover, we emphasize the importance of CV risk factors and stress their impact on disease phenotype in this patient population.