Objective: To explore the symptomatic burden of patients with essential thrombocythemia (ET) and its relation with clinical characteristics including the mutation status, therapeutic protocols and sex.
Methods: Total of 173 Chinese ET patients were selected and grouped on the basis of disease characteristics (mutation status, therapeutic pro to- cols, and sex).
Results: All the groups showed low-to-high symptom burden, with the highest in the Hu (hydroxyurea)-group (total symptom score [TSS], 14.7; range, 7.6-14.7). In the JAK2V617F-positive, Hu-treated, and female groups TSS and independent symptom scores were higher than those in the control group. The CALR-positive and IFN-α-treated groups had lower overall and individual scores as compared with groups lacking the corresponding characteristics. As the number of characteristics (JAK2V617F-positive, Hu-treated, and female) increases, the severity of symptoms gradually increased.
Conclusion: The different characteristics have various effects on symptom burden in ET patients. The accumulation of certain characteristics will lead to more severe symptom burden, thus the patient＇s symptom burden should be considered comprehensively when making up the treatment schemes and prognosis.
题目: 173例中国原发性血小板增多症患者的症状负荷评估.
目的: 探讨中国原发性血小板增多症（essential thrombocythemia，ET）患者的症状负荷与临床特征（突变状态，治疗方案，性别差异）的关系，为其治疗和预后判断提供依据.
方法: 使用骨髓增殖性肿瘤总症状评估量表（MPN-SAF-TSS或MPN 10）对173例中国ET患者进行现场和网络症状评估，根据突变状态将患者分为JAK2V617F突变阳性组，CALR突变阳性组，非三阴性组；根据治疗方案将患者分为羟基脲（Hu）治疗组和干扰素（IFN-α）治疗组；根据性别将患者分为男性患者组和女性患者组。结合症状评分回顾性分析患者的病例资料.
结果: 各组患者均表现出由低至高的症状负荷，其中Hu治疗组患者症状负荷最高（总症状评分[TSS]为14.7；范围7.6-14.7）；与对照组相比，JAK2V617F阳性组、Hu治疗组和女性患者组具有更高的总症状和独立症状评分。 CALR阳性组和IFN-α治疗组的总症状和独立症状得分均低于缺乏相应特征的对照组。JAK2V617F阳性、Hu治疗和女性患者这3个因素的累加会导致更严重的症状负荷.
结论: 中国ET患者的症状负荷与患者的突变状态、治疗选择和性别差异显著相关，制定治疗方案和进行预后判断时应综合考虑患者的症状负荷和相关临床特征.