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慢性髓性白血病的预后:基线因素、动态风险评估和新的见解。

Prognosis in Chronic Myeloid Leukemia: Baseline Factors, Dynamic Risk Assessment and Novel Insights.

机构信息

Dipartimento di Scienze Mediche e Chirurgiche (DIMEC), Università di Bologna, 40138 Bologna, Italy.

Istituto di Ematologia "Seràgnoli", IRCCS Azienda Ospedaliero-Universitaria di Bologna, 40138 Bologna, Italy.

出版信息

Cells. 2023 Jun 23;12(13):1703. doi: 10.3390/cells12131703.

Abstract

The introduction of tyrosine kinase inhibitors (TKIs) has changed the treatment paradigm of chronic myeloid leukemia (CML), leading to a dramatic improvement of the outcome of CML patients, who now have a nearly normal life expectancy and, in some selected cases, the possibility of aiming for the more ambitious goal of treatment-free remission (TFR). However, the minority of patients who fail treatment and progress from chronic phase (CP) to accelerated phase (AP) and blast phase (BP) still have a relatively poor prognosis. The identification of predictive elements enabling a prompt recognition of patients at higher risk of progression still remains among the priorities in the field of CML management. Currently, the baseline risk is assessed using simple clinical and hematologic parameters, other than evaluating the presence of additional chromosomal abnormalities (ACAs), especially those at "high-risk". Beyond the onset, a re-evaluation of the risk status is mandatory, monitoring the response to TKI treatment. Moreover, novel critical insights are emerging into the role of genomic factors, present at diagnosis or evolving on therapy. This review presents the current knowledge regarding prognostic factors in CML and their potential role for an improved risk classification and a subsequent enhancement of therapeutic decisions and disease management.

摘要

酪氨酸激酶抑制剂(TKIs)的引入改变了慢性髓性白血病(CML)的治疗模式,显著改善了 CML 患者的预后,使他们的预期寿命几乎恢复正常,在某些选定的情况下,还有可能实现更具挑战性的无治疗缓解(TFR)目标。然而,少数治疗失败并从慢性期(CP)进展为加速期(AP)和急变期(BP)的患者预后仍然较差。确定能够及时识别具有更高进展风险的患者的预测因素仍然是 CML 管理领域的重点之一。目前,基线风险是使用简单的临床和血液学参数评估的,除了评估是否存在其他染色体异常(ACAs)外,尤其是那些“高危”的异常。在发病后,必须重新评估风险状况,监测 TKI 治疗的反应。此外,在诊断时或治疗过程中出现的基因组因素的作用方面出现了新的重要认识。这篇综述介绍了 CML 中预后因素的最新知识及其在改善风险分类和随后增强治疗决策和疾病管理方面的潜在作用。

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