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<p>Acute myeloid leukemia (AML) is an aggressive hematologic malignancy characterized by dysregulated differentiation and uncontrolled proliferation of myeloid precursor cells. AML is the second most common type of leukemia after acute lymphoblastic leukemia, yet it has the lower survival rates, with only approximately 30% of adult patients surviving five years post-diagnosis. Standard treatment regimens typically include intensive chemotherapy, advances in allogeneic hematopoietic stem cell transplantation (allo-HSCT) have significantly improved outcomes in the treatment of AML. Advances in molecular profiling technologies have significantly enhanced our understanding of the genetic and epigenetic alterations that drive AML, revealing numerous novel therapeutic targets. Consequently, targeted molecular therapies and epigenetic treatments are becoming increasingly important. Moreover, immunotherapy represents a promising therapeutic strategy that has demonstrated considerable potential in the context of AML. This review summarizes new strategies and emerging therapeutic targets in AML, with a particular focus on recent advancements in immunotherapy. It also explores the feasibility of integrating these therapeutic approaches into current treatment paradigms and their potential impact on future clinical practices.</p>

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Emerging strategies and novel therapeutic targets in acute myeloid leukemia: current advances and future directions

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年10月14日

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  • Linyu Cao,
  • Zhe Wang,
  • Yimin Cui,
  • Yuan Li,
  • Qian Xiang

摘要

Acute myeloid leukemia (AML) is an aggressive hematologic malignancy characterized by dysregulated differentiation and uncontrolled proliferation of myeloid precursor cells. AML is the second most common type of leukemia after acute lymphoblastic leukemia, yet it has the lower survival rates, with only approximately 30% of adult patients surviving five years post-diagnosis. Standard treatment regimens typically include intensive chemotherapy, advances in allogeneic hematopoietic stem cell transplantation (allo-HSCT) have significantly improved outcomes in the treatment of AML. Advances in molecular profiling technologies have significantly enhanced our understanding of the genetic and epigenetic alterations that drive AML, revealing numerous novel therapeutic targets. Consequently, targeted molecular therapies and epigenetic treatments are becoming increasingly important. Moreover, immunotherapy represents a promising therapeutic strategy that has demonstrated considerable potential in the context of AML. This review summarizes new strategies and emerging therapeutic targets in AML, with a particular focus on recent advancements in immunotherapy. It also explores the feasibility of integrating these therapeutic approaches into current treatment paradigms and their potential impact on future clinical practices.