Hematology Unit, First Department of Internal Medicine, "Laikon" General Hospital, National and Kapodistrian University of Athens, Athens, Greece,
Hematology Unit, First Department of Internal Medicine, "Laikon" General Hospital, National and Kapodistrian University of Athens, Athens, Greece.
Oncol Res Treat. 2019;42(10):532-535. doi: 10.1159/000502121. Epub 2019 Sep 3.
Leukemic transformation is an unfavorable event emerging in a minority of patients with myelofibrosis (MF) and carrying a poor prognosis. Patients with post-MF acute myeloid leukemia (AML) may be treated with curative or noncurative intent, but there is no standard of care. The use of hypomethylating agents has been correlated with limited activity and low overall survival rates, while ruxolitinib has been proven to have modest antileukemic activity.
In this case study, we present the case of a 67-year-old female patient with post-polycythemia vera MF who developed AML and was successfully treated with a combination of 5-azacytidine and ruxolitinib, without any significant toxicity.
To our knowledge, this is the first report of a patient with post-MF AML achieving a complete remission with a combination of ruxolitinib and a hypomethylating agent. This combination is actively studied in phase I/II clinical trials and may be proven effective in this hard-to-treat group of patients.
白血病转化是少数骨髓纤维化（MF）患者中出现的不利事件，预后不良。继发 MF 的急性髓系白血病（AML）患者可能采用治愈或非治愈意向进行治疗，但尚无标准治疗方案。低甲基化剂的使用与有限的活性和低总生存率相关，而鲁索利替尼已被证明具有适度的抗白血病活性。
在本病例研究中，我们介绍了一位 67 岁女性患者的病例，她继发于真性红细胞增多症后的 MF 发展为 AML，并成功接受了 5-氮杂胞苷和鲁索利替尼联合治疗，无明显毒性。
据我们所知，这是首例报道继发 MF 的 AML 患者采用鲁索利替尼联合低甲基化剂获得完全缓解的病例。该联合方案正在进行 I/II 期临床试验，可能对这一难以治疗的患者群体有效。
