Myeloid blast-phase chronic myeloid leukemia (MBP-CML) is a rare disease with a dismal prognosis. It is twice as common as lymphoid blast-phase CML, and its prognosis is poorer. Despite the success with tyrosine kinase inhibitors in the treatment of chronic-phase CML, the same does not hold true for MBP-CML. In addition to the Philadelphia chromosome, other chromosomal and molecular changes characterize rapid progression. Although some progress in elucidating the biology of MBP-CML has been made, there is need to discover more in order to develop more satisfactory treatment options. Currently, most common treatment options include tyrosine kinase inhibitors (TKIs) as monotherapy or in combination with acute myeloid leukemia-based intensive chemotherapy regimens. Some patients may develop resistance to TKIs via BCR-ABL1-dependent or BCR-ABL1-independent mechanisms. In this paper, we provide an overview of the biology of MBP-CML, the current treatment approaches, and mechanisms of resistance to TKIs. In order to improve treatment responses in these patients, more emphasis should be placed on understanding the biology of myeloid blastic transformation in CML and mechanisms of resistance to TKIs. Although patient numbers are small, randomized clinical trials should be considered.
髓系急变期慢性粒细胞白血病（MBP-CML）是一种预后极差的罕见疾病。其发病率约为淋巴系急变期CML的两倍，且预后更为不良。尽管酪氨酸激酶抑制剂在慢性期CML治疗中取得显著成效，但该疗效并未延伸至MBP-CML领域。除费城染色体外，其他染色体与分子层面的改变共同构成了疾病快速进展的特征。虽然目前对MBP-CML生物学机制的研究已取得一定进展，但仍需进一步深入探索以开发更理想的治疗方案。当前主流治疗策略包括酪氨酸激酶抑制剂单药治疗，或联合基于急性髓系白血病方案的强化化疗。部分患者可能通过BCR-ABL1依赖性或非依赖性机制产生TKI耐药性。本文系统综述了MBP-CML的生物学特征、现行治疗策略及TKI耐药机制。为提升此类患者的治疗应答率，应着重深化对CML髓系急变生物学机制及TKI耐药途径的理解。尽管患者群体规模有限，仍应考虑开展随机临床试验。
肿瘤(癌症)患者之家