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一名对酪氨酸激酶抑制剂治疗持续保持主要分子学反应的慢性粒细胞白血病患者,出现费城染色体阴性急性髓系白血病的快速进展。

Rapid development of Philadelphia chromosome-negative AML in a CML patient with sustained major molecular response to tyrosine kinase inhibitor therapy.

作者信息

Liu Huan, Sun Yunxia, Li Liangliang, Zhang Yurong, Zhou Yaxiong, Zeng Pengyun, Yue Lingling

机构信息

Department of Hematology, The Second Hospital & Clinical Medical School, Lanzhou University, No. 82, Cuyingmen, Lanzhou, Gansu Province 730030, China.

Department of Hematology, The First People's Hospital of Lanzhou, China.

出版信息

Leuk Res Rep. 2025 Aug 21;24:100536. doi: 10.1016/j.lrr.2025.100536. eCollection 2025.

Abstract

The use of TKIs has significantly improved the prognosis of CML. However, a small subset of patients still experience poor outcomes. We present a rare case of Ph-AML following a diagnosis of CML. The patient achieved CCyR and MMR after 4 months of nilotinib therapy, with sustained deep remission for 3 years. Unexpectedly, the disease developed rapidly to AML. Further investigations revealed the emergence of CCA/Ph- and gene mutations. We retrospectively analyzed previous CML patients with and Ph-negative status in blast crisis from our database and conducted a comprehensive review of the relevant literature.

摘要

酪氨酸激酶抑制剂(TKIs)的使用显著改善了慢性粒细胞白血病(CML)的预后。然而,一小部分患者的预后仍然较差。我们报告了一例诊断为CML后发生Ph阴性急性髓系白血病(Ph-AML)的罕见病例。患者在接受尼洛替尼治疗4个月后达到完全细胞遗传学缓解(CCyR)和主要分子学缓解(MMR),并持续深度缓解3年。出乎意料的是,疾病迅速发展为AML。进一步检查发现了CCA/Ph-和基因突变的出现。我们回顾性分析了我们数据库中既往处于急变期且具有和Ph阴性状态的CML患者,并对相关文献进行了全面综述。

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