Hu Bei, Vikas Praveen, Mohty Mohamad, Savani Bipin N
Department of Medicine, Vanderbilt University Medical Center, Nashville, TN, USA.
Expert Rev Hematol. 2014 Apr;7(2):301-15. doi: 10.1586/17474086.2014.857596. Epub 2013 Dec 6.
Survival of patients with acute myelogenous leukemia (AML), particularly in younger patients, has improved in recent years due to improved understanding of disease biology, post remission therapies and supportive care. AML, however, remains difficult to treat as many patients will still ultimately relapse and die of their disease. This is particularly true in AML patients with identified FMS-like tyrosine kinase 3-internal tandem duplication (FLT3-ITD) molecular mutations, which typically confers a poor prognosis. The FLT3-ITD mutation occurs in about one-quarter of patients diagnosed with AML. Oftentimes, these patients are referred for early allogeneic hematopoietic stem cell transplantation (HSCT) in hopes of overcoming this poor prognostic factor. Several studies have demonstrated some benefit with HSCT in patients with FLT3-ITD mutation. However, recent data suggested that FLT3-ITD mutation remains a poor prognostic factor even after early HSCT; these patients remain at risk for early relapse after transplantation, emphasizing ongoing efforts to explore maintenance therapy with FLT3-ITD inhibitors in the post-transplant setting.
近年来，由于对疾病生物学、缓解后治疗和支持性护理的认识有所提高，急性髓系白血病（AML）患者，尤其是年轻患者的生存率有所改善。然而，AML仍然难以治疗，因为许多患者最终仍会复发并死于该疾病。在确诊为FMS样酪氨酸激酶3-内部串联重复（FLT3-ITD）分子突变的AML患者中尤其如此，这种突变通常预示着预后不良。FLT3-ITD突变发生在约四分之一的AML确诊患者中。通常，这些患者会被转诊接受早期异基因造血干细胞移植（HSCT），以期克服这一不良预后因素。多项研究已证明HSCT对FLT3-ITD突变患者有一定益处。然而，近期数据表明，即使在早期HSCT后，FLT3-ITD突变仍是一个不良预后因素；这些患者在移植后仍有早期复发的风险，这凸显了在移植后环境中探索使用FLT3-ITD抑制剂进行维持治疗的持续努力。
