Zhou Lu, Shi Huiping, Shi Wenyu, Yang Li, Zhang Yaping, Xu Mengqi, Chen Xiufang, Zhu Yanv, Mu Hui, Wan Xiaochun, Yang Zhonghua, Zeng Ying, Liu Hong
Hematology Department, Affiliated Hospital of Nantong University, Nantong, People's Republic of China.
Cardiovascular Biology Research Program, Oklahoma Medical Research Foundation, Oklahoma City, Oklahoma, USA.
Onco Targets Ther. 2019 Dec 13;12:10989-10995. doi: 10.2147/OTT.S232102. eCollection 2019.
Despite the prominent effects of BCR-ABL tyrosine kinase inhibitors (TKI) therapy in patients with chronic phase-chronic myeloid leukemia (CP-CML) and thus low incidence of blastic transformation, blast phase (BP)-CML remains a major therapeutic challenge in the TKI era. The "gatekeeper" mutation T315I in BCR-ABL1 kinase, which often coupled with a poor prognosis, is quite common and resistant to all TKIs except for ponatinib. The occurrence of T315I mutation in BP-CML makes the situation more complex. Anti-CD19 chimeric antigen receptor T cell (CAR-T) technology is a new immunotherapy which has significantly improved the efficacy of B cell hematologic malignances. Here we report a lymphoid BP-CML patient harboring T315I mutation who achieved complete molecular remission and returned to chronic phase by anti-CD19 CAR-T therapy. Our study provides a new therapeutic strategy for patients in BP-CML.
尽管BCR-ABL酪氨酸激酶抑制剂（TKI）疗法对慢性期慢性髓性白血病（CP-CML）患者有显著疗效，且急变期发生率较低，但急变期（BP）-CML在TKI时代仍然是一个主要的治疗挑战。BCR-ABL1激酶中的“守门人”突变T315I通常与不良预后相关，十分常见，并且除波纳替尼外对所有TKI均耐药。BP-CML中T315I突变的出现使情况更加复杂。抗CD19嵌合抗原受体T细胞（CAR-T）技术是一种新型免疫疗法，已显著提高了B细胞血液系统恶性肿瘤的疗效。在此，我们报告1例携带T315I突变的淋巴系BP-CML患者，通过抗CD19 CAR-T疗法实现了完全分子缓解并恢复到慢性期。我们的研究为BP-CML患者提供了一种新的治疗策略。
