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骨髓增殖性肿瘤症状评估总症状评分(MPN-SAF TSS)与遗传负担和血栓形成在慢性骨髓增殖性肿瘤中的关系。

Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score (MPN-SAF TSS) in Chronic Myeloproliferative Neoplasms with Relation to Genetic Burden and Thrombosis.

机构信息

Trakya University Faculty of Medicine, Department of Internal Medicine, Division of Hematology, Edirne, Türkiye

Ali Osman Sönmez Oncology Hospital, Clinic of Hematology, Bursa, Türkiye

出版信息

Turk J Haematol. 2024 Aug 28;41(3):175-181. doi: 10.4274/tjh.galenos.2024.2024.0011. Epub 2024 May 27.

Abstract

The Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score (MPN-SAF TSS) is a surrogate marker for symptom evaluation in chronic myeloproliferative neoplasms (MPNs). However, insufficient data are available regarding the relationship among the MPN-SAF TSS, mutation allele burden, and thrombosis. In this retrospective analysis, we aimed to determine the genetic burdens, clinical features, and relationships with MPN-SAF TSS in MPN patients. One hundred thirty V617F-positive patients with MPNs were included in our study. We calculated the MPN-SAF TSS for all patients and compared it with their clinical characteristics. Patients with higher V617F mutation allele burden had higher MPN-SAF TSS values (p=0.008). Patients with thrombosis had higher MPN-SAF TSS than patients without thrombosis (p=0.003). The mean MPN-SAF TSS was higher in patients with primary myelofibrosis compared to those with polycythemia vera and essential thrombocythemia. Thrombosis was associated with increased symptom severity in several domains, including fatigue, abdominal discomfort, inactivity, night sweats, pruritus, weight loss, and early satiety. Additionally, an increase in allele burden was observed with higher symptom scores. The MPN-SAF TSS proved to be a reliable tool for assessing symptom burden in Turkish MPN patients. Furthermore, the significant association between thrombosis occurrence and symptom severity suggests that thrombotic events may contribute to symptom development. Notably, increasing allele burden was correlated with more severe symptoms, highlighting its potential role in predicting disease burden. This study emphasizes the importance of symptom assessment in MPN patients and supports the incorporation of the MPN-SAF TSS in routine clinical practice to enhance patient care and management.

摘要

骨髓增殖性肿瘤症状评估总症状评分(MPN-SAF TSS)是一种用于评估慢性骨髓增殖性肿瘤(MPN)患者症状的替代标志物。然而,关于 MPN-SAF TSS、突变等位基因负担与血栓形成之间的关系,目前数据还不够充分。在这项回顾性分析中,我们旨在确定 MPN 患者的基因负担、临床特征及其与 MPN-SAF TSS 的关系。我们纳入了 130 例 V617F 阳性 MPN 患者,计算了所有患者的 MPN-SAF TSS,并将其与临床特征进行了比较。突变等位基因负担较高的患者,其 MPN-SAF TSS 值更高(p=0.008)。有血栓形成的患者的 MPN-SAF TSS 高于无血栓形成的患者(p=0.003)。与原发性骨髓纤维化患者相比,真性红细胞增多症和原发性血小板增多症患者的 MPN-SAF TSS 平均值更高。血栓形成与多个领域的症状严重程度增加有关,包括疲劳、腹部不适、活动减少、夜间出汗、瘙痒、体重减轻和早饱。此外,随着症状评分的增加,等位基因负担也有所增加。MPN-SAF TSS 被证明是评估土耳其 MPN 患者症状负担的可靠工具。此外,血栓形成的发生与症状严重程度之间的显著关联表明,血栓事件可能导致症状的发生。值得注意的是,等位基因负担的增加与更严重的症状相关,这突出了其在预测疾病负担方面的潜在作用。本研究强调了对 MPN 患者进行症状评估的重要性,并支持在常规临床实践中纳入 MPN-SAF TSS,以提高患者的护理和管理水平。

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