Song Baoquan, Wang Xin, Kong Xin, Wang Man, Yao Li, Shen Hongjie, Zhang Jian, Qiu Huiying
National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China.
Institute of Blood and Marrow Transplantation, Collaborative Innovation Center of Hematology, Soochow University, Suzhou, China.
Front Oncol. 2022 Feb 7;12:828852. doi: 10.3389/fonc.2022.828852. eCollection 2022.
Variant acute promyelocytic leukemia (APL) showed quite different aspects, and the current treatments remained challenged at present. Venetoclax, a selective inhibitor of B-cell lymphoma 2 (BCL-2), is a small molecule that has been studied in several hematologic malignancies as both monotherapy and in combination with other agents. However, there is little of its use in the treatment of APL or variant APL. In this report, we identified THRAP3 as novel RARA fusion in resembling APL, which was resistant to all-trans retinoic acid (ATRA) combined arsenic trioxide (ATO) chemotherapy. Then, the patient was salvaged by low-dose venetoclax and decitabine. The treatment in this case demonstrates the potential ability of venetoclax in variant APL, thus providing a new treatment option for all kinds of APL.
变异型急性早幼粒细胞白血病（APL）表现出截然不同的情况，目前的治疗方法仍然面临挑战。维奈克拉是一种B细胞淋巴瘤2（BCL-2）的选择性抑制剂，是一种小分子药物，已在多种血液系统恶性肿瘤中作为单一疗法以及与其他药物联合使用进行了研究。然而，其在治疗APL或变异型APL方面的应用很少。在本报告中，我们在类似APL的病例中鉴定出THRAP3为新型RARA融合基因，该病例对全反式维甲酸（ATRA）联合三氧化二砷（ATO）化疗耐药。然后，该患者通过低剂量维奈克拉和地西他滨得到挽救。该病例的治疗证明了维奈克拉在变异型APL中的潜在作用，从而为各类APL提供了一种新的治疗选择。
