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对于无法进行异基因造血干细胞移植的急性髓系白血病缓解期患者诱导缓解后维持治疗的临床观点

Clinical perspectives on post-induction maintenance therapy in patients with acute myeloid leukaemia in remission who are ineligible for allogeneic haematopoietic stem cell transplantation.

作者信息

Sweet Kendra, Cluzeau Thomas

机构信息

H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida, USA.

Université Cote d'Azur, CHU de Nice, Service d'hématologie, Nice, France.

出版信息

Br J Haematol. 2025 Jan;206(1):61-68. doi: 10.1111/bjh.19924. Epub 2024 Dec 2.

Abstract

For patients with acute myeloid leukaemia (AML) who achieve complete remission (CR) after induction therapy, subsequent allogeneic haematopoietic stem cell transplantation (allo-HSCT) reduces the risk of relapse. However, not all patients are eligible, warranting effective alternative maintenance strategies. Oral azacitidine is the only non-targeted therapy approved by both the United States (US) Food and Drug Administration and the European Medicines Agency for the maintenance or continued treatment of allo-HSCT-ineligible patients with AML achieving CR or CR with incomplete haematological recovery following induction chemotherapy. Midostaurin and histamine dihydrochloride are approved in Europe as maintenance therapy for AML in remission, and quizartinib is approved in the United States and Europe for the treatment and maintenance of patients with newly diagnosed FLT3-ITD AML. Barriers to maintenance treatment include limited clinical trial data informing appropriate patient and treatment selection, patient preference, financial burden and paucity of real-world data. This article discusses current maintenance treatment guidelines for patients with AML in remission but not proceeding to allo-HSCT and reviews clinical trial data for agents approved for use in remission. Ongoing studies of interest and considerations for future efforts are also discussed.

摘要

对于诱导治疗后达到完全缓解(CR)的急性髓系白血病(AML)患者,后续的异基因造血干细胞移植(allo-HSCT)可降低复发风险。然而,并非所有患者都符合条件,因此需要有效的替代维持策略。口服阿扎胞苷是唯一经美国食品药品监督管理局和欧洲药品管理局批准用于维持治疗或继续治疗不符合allo-HSCT条件、在诱导化疗后达到CR或血液学未完全恢复的CR的AML患者的非靶向治疗药物。米哚妥林和盐酸组胺在欧洲被批准作为AML缓解期的维持治疗药物,而 quizartinib在美国和欧洲被批准用于新诊断的FLT3-ITD AML患者的治疗和维持治疗。维持治疗的障碍包括用于指导合适患者和治疗选择的临床试验数据有限、患者偏好、经济负担以及真实世界数据匮乏。本文讨论了未进行allo-HSCT的AML缓解期患者的当前维持治疗指南,并回顾了已批准用于缓解期的药物的临床试验数据。还讨论了正在进行的相关研究以及对未来工作的考虑。

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