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酪氨酸激酶抑制剂时代慢性髓性白血病急变期患者的管理和预后 - 欧洲白血病网急变期登记处分析。

Management and outcome of patients with chronic myeloid leukemia in blast phase in the tyrosine kinase inhibitor era - analysis of the European LeukemiaNet Blast Phase Registry.

机构信息

Klinik und Poliklinik für Innere Medizin C, Hämatologie und Onkologie, Universitätsmedizin Greifswald, Greifswald, Germany.

Klinik für Innere Medizin II, Universitätsklinikum Jena, Comprehensive Cancer Center Central Germany, Campus Jena, Jena, Germany.

出版信息

Leukemia. 2024 May;38(5):1072-1080. doi: 10.1038/s41375-024-02204-y. Epub 2024 Mar 28.

Abstract

Blast phase (BP) of chronic myeloid leukemia (CML) still represents an unmet clinical need with a dismal prognosis. Due to the rarity of the condition and the heterogeneity of the biology and clinical presentation, prospective trials and concise treatment recommendations are lacking. Here we present the analysis of the European LeukemiaNet Blast Phase Registry, an international collection of the clinical presentation, treatment and outcome of blast phases which had been diagnosed in CML patients after 2015. Data reveal the expected heterogeneity of the entity, lacking a clear treatment standard. Outcomes remain dismal, with a median overall survival of 23.8 months (median follow up 27.8 months). Allogeneic stem cell transplantation (alloSCT) increases the rate of deep molecular responses. De novo BP and BP evolving from a previous CML do show slightly different features, suggesting a different biology between the two entities. Data show that outside clinical trials and in a real-world setting treatment of blast phase is individualized according to disease- and patient-related characteristics, with the aim of blast clearance prior to allogeneic stem cell transplantation. AlloSCT should be offered to all patients eligible for this procedure.

摘要

慢性髓性白血病(CML)的急变期仍然是一个未满足的临床需求,预后较差。由于该疾病的罕见性以及生物学和临床表现的异质性,缺乏前瞻性试验和简明的治疗建议。在这里,我们分析了欧洲白血病网急变期登记处,这是一个国际收集的 2015 年后诊断为 CML 患者的急变期临床表现、治疗和结局的登记处。数据揭示了该实体的预期异质性,缺乏明确的治疗标准。结果仍然很差,中位总生存期为 23.8 个月(中位随访时间为 27.8 个月)。异基因造血干细胞移植(alloSCT)可提高深度分子反应率。新发急变期和由先前 CML 演变而来的急变期确实表现出略有不同的特征,表明这两种实体之间存在不同的生物学。数据表明,在临床试验之外的真实环境中,急变期的治疗是根据疾病和患者相关特征个体化的,目的是在异基因造血干细胞移植前清除急变细胞。所有符合条件的患者都应接受 alloSCT。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/871f/11073984/66a383ca1bc7/41375_2024_2204_Fig1_HTML.jpg

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