文章：

真实世界电子病历数据揭示骨髓纤维化风险因素，并可用于验证既定预后评分系统

Real-World Electronic Medical Records Data Identify Risk Factors for Myelofibrosis and Can Be Used to Validate Established Prognostic Scores

原文发布日期：5 April 2024

DOI: 10.3390/cancers16071416

类型: Article

开放获取: 是

英文摘要：

Myelofibrosis (MF) is a myeloproliferative neoplasia arising de novo as primary myelofibrosis (PMF) or secondary to polycythemia vera or essential thrombocythemia. Patients experience a high symptom burden and a marked reduction in life expectancy. Despite progress in molecular understanding and treatment, the clinical and prognostic heterogeneity of MF complicates treatment decisions. The International Prognostic Scoring System (IPSS) integrates clinical factors for risk stratification in MF. This study leverages the TriNetX database with more than 64,000 MF patients to assess the impact of accessible parameters on survival and complicating events, including AML transformation, cachexia, increased systemic inflammation, thrombosis and hemorrhage. Age over 65 years correlated with increased risks of death, AML transformation, thrombosis and hemorrhage. Anemia (Hb < 10 g/dL), leukocytosis (>25 × 103/µL) and thrombocytopenia (<150 × 103/µL) reduced survival and increased risks across all assessed events. Monocytosis is associated with decreased survival, whereas eosinophilia and basophilia were linked to improved survival. Further, as proof of concept for the applicability of TriNetX for clinical scores, we devised a simplified IPSS, and confirmed its value in predicting outcomes. This comprehensive study underscores the importance of age, anemia, leukocytosis and thrombocytopenia in predicting disease trajectory and contributes to refining prognostic models, addressing the challenges posed by the disease’s heterogeneity.

摘要翻译： 

骨髓纤维化（MF）是一种骨髓增殖性肿瘤，可原发为原发性骨髓纤维化（PMF），也可继发于真性红细胞增多症或原发性血小板增多症。患者症状负担重，预期寿命显著缩短。尽管在分子机制理解和治疗方面取得进展，但MF的临床和预后异质性使治疗决策复杂化。国际预后评分系统（IPSS）整合临床因素对MF进行风险分层。本研究利用包含超过64,000名MF患者的TriNetX数据库，评估可获取参数对生存及并发症事件（包括急性髓系白血病转化、恶病质、全身炎症加重、血栓形成和出血）的影响。年龄超过65岁与死亡、AML转化、血栓形成和出血风险增加相关。贫血（Hb < 10 g/dL）、白细胞增多（>25 × 10³/µL）和血小板减少（<150 × 10³/µL）会降低生存率，并增加所有评估事件的风险。单核细胞增多与生存率降低相关，而嗜酸性粒细胞增多和嗜碱性粒细胞增多则与生存改善相关。此外，作为TriNetX应用于临床评分可行性的概念验证，我们设计了一个简化的IPSS，并证实其在预测结局方面的价值。这项综合性研究强调了年龄、贫血、白细胞增多和血小板减少在预测疾病进展轨迹中的重要性，有助于完善预后模型，应对疾病异质性带来的挑战。

原文链接：

Real-World Electronic Medical Records Data Identify Risk Factors for Myelofibrosis and Can Be Used to Validate Established Prognostic Scores