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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
Leukemia ( IF 11.4 ) Pub Date : 2024-03-28 , DOI: 10.1038/s41375-024-02204-y
Annamaria Brioli , Elza Lomaia , Christian Fabisch , Tomasz Sacha , Hana Klamova , Elena Morozova , Aleksandra Golos , Philipp Ernst , Ulla Olsson-Stromberg , Daniela Zackova , Franck E. Nicolini , Han Bao , Fausto Castagnetti , Elzbieta Patkowska , Jiri Mayer , Klaus Hirschbühl , Helena Podgornik , Edyta Paczkowska , Anne Parry , Thomas Ernst , Astghik Voskanyan , Elzbieta Szczepanek , Susanne Saussele , Georg-Nikolaus Franke , Alexander Kiani , Edgar Faber , Stefan Krause , Luis Felipe Casado , Krzysztof Lewandowski , Matthias Eder , Peter Anhut , Justyna Gil , Thomas Südhoff , Holger Hebart , Sonja Heibl , Markus Pfirrmann , Andreas Hochhaus , Michael Lauseker

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.



中文翻译:

酪氨酸激酶抑制剂时代慢性粒细胞白血病急变期患者的管理和结果——欧洲 LeukemiaNet 急变期登记分析

慢性粒细胞白血病 (CML) 的急变期 (BP) 仍然代表着未满足的临床需求,且预后不佳。由于该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。在此,我们介绍了欧洲 LeukemiaNet 急变期登记处的分析,该登记处收集了 2015 年之后在 CML 患者中诊断出的急变期的临床表现、治疗和结果。数据揭示了该实体的预期异质性,缺乏明确的治疗方法标准。结果仍然令人沮丧,中位总生存期为 23.8 个月(中位随访时间 27.8 个月)。同种异体干细胞移植(alloSCT)可提高深层分子反应的速度。 De novo BP 和从之前的 CML 演变而来的 BP 确实表现出略有不同的特征,表明这两个实体之间存在不同的生物学特性。数据显示,在临床试验之外和现实环境中,原始细胞期的治疗是根据疾病和患者相关特征进行个体化的,目的是在同种异体干细胞移植之前清除原始细胞。应向所有符合该手术资格的患者提供 AlloSCT。

更新日期:2024-03-29
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