Yousefi Arjen, Levin Mark-David, Cornelissen Jan J, Westerweel Peter E
Department of internal medicine, Albert Schweitzer Hospital, Dordrecht, The Netherlands.
Department of hematology, Erasmus Medical Center, Rotterdam, The Netherlands.
Ann Hematol. 2025 Mar;104(3):2065-2068. doi: 10.1007/s00277-024-06170-4. Epub 2025 Jan 10.
Selected chronic myeloid leukemia (CML) patients may discontinue their tyrosine kinase inihibitor (TKI) in an attempt to achieve sustained treatment-free remission (TFR), which mitigates therapy-related side effects and limits treatment costs. TFR has been extensively studied following the discontinuation of adenosine triphosphate (ATP) - competitive TKI. However, there is minimal data concerning TFR after the discontinuation of the novel TKI asciminib. Here, we present two CML patients intolerant to multiple ATP-competitive TKIs who achieved a deep molecular response (DMR) during asciminib treatment and sustained this remission after asciminib discontinuation. One of the cases developed transient myalgia and arthralgia after asciminib discontinuation consistent with a TKI withdrawal syndrome. Both patients have been free of molecular relapse for more than at least 8 months after TKI discontinuation without increase in molecular BCR::ABL1 signal. These two cases provide proof of principle that sustained TFR after discontinuing asciminib in CML patients is feasible.
部分慢性髓性白血病（CML）患者可能会停用酪氨酸激酶抑制剂（TKI），以期实现持续的无治疗缓解（TFR），这可减轻治疗相关副作用并降低治疗成本。在停用三磷酸腺苷（ATP）竞争性TKI后，对TFR已进行了广泛研究。然而，关于新型TKI阿伐替尼停用后的TFR数据极少。在此，我们报告了两名对多种ATP竞争性TKI不耐受的CML患者，他们在阿伐替尼治疗期间实现了深度分子反应（DMR），并在停用阿伐替尼后维持了这种缓解状态。其中一例患者在停用阿伐替尼后出现了短暂的肌痛和关节痛，符合TKI撤药综合征。两名患者在停用TKI后均至少8个月无分子复发，且分子BCR::ABL1信号未增加。这两个病例提供了原理证明，即CML患者停用阿伐替尼后实现持续TFR是可行的。
