近年来,慢性髓性白血病(CML)的治疗方法发生了变化。因此,目前,处于慢性期的患者中有很高的比例几乎具有平均预期寿命。治疗还旨在实现稳定的深层分子反应(DMR),这可能允许剂量减少或甚至停止治疗。这些策略通常用于真实的实践,以减少不良事件,然而,它们对免治疗缓解(TFR)的影响仍存在争议.在一些研究中,据观察,多达一半的患者在停止TKI治疗后可达到TFR.如果TFR更广泛且在全球范围内可实现,对毒性的看法可以改变。我们回顾性分析了2002年至2022年间在三级医院接受酪氨酸激酶抑制剂(TKI)治疗的80例CML患者。从他们那里,71例患者接受低剂量TKI治疗,25个最终停产,其中9个在没有先前剂量减少的情况下被停用。关于低剂量治疗的患者,其中只有11例出现分子复发(15.4%),平均分子无复发生存期(MRFS)为24.6个月。MRFS结果不受任何检查变量的影响,包括性别,索卡尔风险评分,之前用干扰素或羟基脲治疗,CML诊断时的年龄,低剂量治疗的开始和TKI治疗的平均持续时间。TKI停药后,除四名患者外,所有患者均保持MMR,中位随访时间为29.2个月。在我们的研究中,TFR估计为38.9个月(95%CI4.1-73.9)。这项研究表明,低剂量治疗和/或TKI停药是一个显著的,对于可能遭受不良事件(AE)的患者,应考虑安全的替代方案,这阻碍了TKI的依从性和/或降低了他们的生活质量。连同已出版的文献,研究表明,减少CML慢性期患者的剂量似乎是安全的.一旦达到DMR,就停止TKI治疗是这些患者的目标之一。应该对患者进行全球评估,并应考虑最合适的管理策略。未来的研究需要确保这种方法被纳入临床实践,因为对某些患者的好处和提高医疗系统的效率。
The therapeutic approach to chronic myeloid leukaemia (CML) has changed in recent years. As a result, a high percentage of current patients in the chronic phase of the disease almost have an average life expectancy. Treatment also aims to achieve a stable deep molecular response (DMR) that might allow dose reduction or even treatment discontinuation. These strategies are often used in authentic practices to reduce adverse events, yet their impact on treatment-free remission (TFR) is a controversial debate. In some studies, it has been observed that as many as half of patients can achieve TFR after the discontinuation of TKI treatment. If TFR was more widespread and globally achievable, the perspective on toxicity could be changed. We retrospectively analysed 80 CML patients treated with tyrosine kinase inhibitor (TKI) at a tertiary hospital between 2002 and 2022. From them, 71 patients were treated with low doses of TKI, and 25 were eventually discontinued, 9 of them being discontinued without a previous dose reduction. Regarding patients treated with low doses, only 11 of them had molecular recurrence (15.4%), and the average molecular recurrence free survival (MRFS) was 24.6 months. The MRFS outcome was not affected by any of the variables examined, including gender, Sokal risk scores, prior treatment with interferon or hydroxycarbamide, age at the time of CML diagnosis, the initiation of low-dose therapy and the mean duration of TKI therapy. After TKI discontinuation, all but four patients maintained MMR, with a median follow-up of 29.2 months. In our
study, TFR was estimated at 38.9 months (95% CI 4.1-73.9). This
study indicates that low-dose treatment and/or TKI discontinuation is a salient, safe alternative to be considered for patients who may suffer adverse events (AEs), which hinder the adherence of TKI and/or deteriorate their life quality. Together with the published literature, it shows that it appears safe to administer reduced doses to patients with CML in the chronic phase. The discontinuation of TKI therapy once a DMR has been reached is one of the goals for these patients. The patient should be assessed globally, and the most appropriate strategy for management should be considered. Future studies are needed to ensure that this approach is included in clinical practice because of the benefits for certain patients and the increased efficiency for the healthcare system.