Zhang Xuzhao, Chen Jinliang, Wang Weiqin, Li Xian, Tan Yanbin, Zhang Xiaohong, Qian Wenbin
Department of Hematology, The Second Affiliated Hospital of Zhejiang University School of Medicine, Hangzhou, China.
Center of Clinical Pharmacology, The Second Affiliated Hospital of Zhejiang University School of Medicine, Hangzhou, China.
Front Oncol. 2021 Jul 5;11:693670. doi: 10.3389/fonc.2021.693670. eCollection 2021.
Extramedullary relapse of acute promyelocytic leukemia is a rare phenomenon and is associated with a poor prognosis, with the central nervous system being the most common site of relapse. The current treatments are still limited. Venetoclax, a selective inhibitor of BCL2, is a small molecule that can cross the blood-brain barrier and shows a potential efficacy in the treatment of chronic lymphocytic leukemia with central nervous system involvement. Although venetoclax has also been used in the treatment of acute myeloid leukemia in recent years, there are no reports of its use in the treatment of central nervous system relapse in acute promyelocytic leukemia. Here, we report a case of central nervous system relapse in acute promyelocytic leukemia that achieved complete remission after oral treatment with venetoclax. The presence of venetoclax in the patient's CSF was confirmed by testing CSF and plasma by mass spectrometry. The concentration of venetoclax in CSF was approximately 1/300 of that in plasma trough concentration. The treatment experience in this case demonstrates the potential ability of venetoclax to treat of central nervous system relapse/involvement in acute promyelocytic leukemia, thus providing a new treatment option for this kind of patient.
急性早幼粒细胞白血病的髓外复发是一种罕见现象，且预后较差，中枢神经系统是最常见的复发部位。目前的治疗方法仍然有限。维奈克拉是一种BCL2选择性抑制剂，是一种能穿过血脑屏障的小分子，在治疗伴有中枢神经系统受累的慢性淋巴细胞白血病方面显示出潜在疗效。尽管近年来维奈克拉也被用于治疗急性髓系白血病，但尚无其用于治疗急性早幼粒细胞白血病中枢神经系统复发的报道。在此，我们报告1例急性早幼粒细胞白血病中枢神经系统复发患者，经口服维奈克拉治疗后实现完全缓解。通过质谱法检测脑脊液和血浆证实患者脑脊液中存在维奈克拉。脑脊液中维奈克拉的浓度约为血浆谷浓度的1/300。该病例的治疗经验证明了维奈克拉治疗急性早幼粒细胞白血病中枢神经系统复发/受累的潜在能力，从而为这类患者提供了一种新的治疗选择。
