Division of Hematological Malignancies and Stem Cell Transplantation, Department of Oncology, The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University, 1650 Orleans Street, Baltimore, MD 21287, USA.
Leukemia Service, Department of Medicine, Memorial Sloan Kettering, 530 East 74th Street, New York, NY 10021, USA. Electronic address: https://twitter.com/RaajitRampal.
Hematol Oncol Clin North Am. 2021 Apr;35(2):325-335. doi: 10.1016/j.hoc.2020.12.008. Epub 2021 Feb 3.
Accelerated and blast phase myeloproliferative neoplasms are advanced stages of the disease with historically a poor prognosis and little improvement in outcomes thus far. The lack of responses to standard treatments likely results from the more aggressive biology reflected by the higher incidence of complex karyotype and high-risk somatic mutations, which are enriched at the time of transformation. Treatment options include induction chemotherapy (7 + 3) as that used on de novo acute myeloid leukemia or hypomethylating agent-based therapy, which has shown similar outcomes. Allogeneic stem cell transplantation remains the only potential for cure.
加速期和急变期骨髓增生性肿瘤是疾病的晚期阶段，历史上预后较差，迄今为止疗效改善甚微。对标准治疗的反应不佳可能是由于更具侵袭性的生物学特性所致，表现为复杂核型和高危体细胞突变的发生率更高，这些突变在转化时更为丰富。治疗选择包括诱导化疗（7+3），如用于初发急性髓系白血病的方案，或基于低甲基化剂的治疗，其疗效相似。异基因造血干细胞移植仍然是唯一可能治愈的方法。
