Bourne Garrett, Bhatia Ravi, Jamy Omer
Department of Medicine, University of Alabama at Birmingham, Birmingham, AL 35294, USA.
J Clin Med. 2024 Apr 27;13(9):2567. doi: 10.3390/jcm13092567.
With the discovery of tyrosine kinase inhibitors (TKIs), overall survival in patients with chronic myeloid leukemia (CML) now approaches that of the general population. While these TKIs have proven to be lifesaving, remaining on them lifelong creates both physical and financial burdens for patients. Recently, multiple trials have begun looking into the efficacy of trialing patients off these TKIs to see if they can sustain treatment-free remission (TFR). TFR eligibility is currently limited to a small population of patients with both robust and sustained responses to TKIs. Currently, for those who attempt a trial of TFR, the average success rates are promising, with anywhere from 38 to 54% of patients experiencing sustained TFR. For those who fail to maintain sustained TFR, safety results to date are reassuring, with almost all patients successfully responding to the re-initiation of TKIs, with death and disease progression being very rare complications. Moving forward, research is being conducted to more accurately risk stratify patients at diagnosis and pair them with optimized upfront treatment regimens aimed at increasing candidacy for the trial of TFR.
随着酪氨酸激酶抑制剂(TKIs)的发现,慢性髓性白血病(CML)患者的总生存期现已接近普通人群。虽然这些TKIs已被证明能挽救生命,但患者终身服用会给他们带来身体和经济负担。最近,多项试验已开始研究让患者停用这些TKIs的疗效,以观察他们是否能维持无治疗缓解(TFR)。目前,TFR的适用对象仅限于一小部分对TKIs有强烈且持续反应的患者。目前,对于那些尝试TFR试验的患者来说,平均成功率很可观,有38%至54%的患者实现了持续的TFR。对于那些未能维持持续TFR的患者,迄今为止的安全性结果令人放心,几乎所有患者在重新开始服用TKIs后都成功产生反应,死亡和疾病进展是非常罕见的并发症。展望未来,正在进行研究,以便在诊断时更准确地对患者进行风险分层,并为他们匹配优化的初始治疗方案,旨在增加TFR试验的入选资格。