Wang Fuping, Qiu Tian, Wang Haitao, Yang Qiong
Beijing Key Laboratory of Resistant Gene Resources and Molecular Development, College of Life Sciences, Beijing Normal University, Beijing, China.
Department of Pathology, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
Clin Lymphoma Myeloma Leuk. 2022 May;22(5):e350-e362. doi: 10.1016/j.clml.2021.11.007. Epub 2021 Nov 15.
Myelofibrosis (MF) is a BCR-ABL1-negative myeloproliferative neoplasm characterized by anemia, extramedullary hematopoiesis, bone marrow fibrosis, splenomegaly, constitutional symptoms and acute myeloid leukemia progression. Currently, allogeneic haematopoietic stem cell transplantation (AHSCT) therapy is the only curative option for MF patients. However, AHSCT is strictly limited due to the high rates of morbidity and mortality. Janus kinase 2 (JAK2) inhibitor Ruxolitinib is the first-line treatment for intermediate-II or high-risk MF patients with splenomegaly and constitutional symptoms, but most MF patients develop resistance or intolerance to Ruxolitinib. Therefore, MF treatment is a challenge for the medical community. This review summarizes 3 investigated directions for MF therapy: monotherapies of JAK inhibitors, monotherapies of non-JAK targeted agents, combination therapies of Ruxolitinib and other agents. We emphasize combination of Ruxolitinib and other agents is a promising strategy.
骨髓纤维化（MF）是一种BCR-ABL1阴性骨髓增殖性肿瘤，其特征为贫血、髓外造血、骨髓纤维化、脾肿大、全身症状以及急性髓系白血病进展。目前，异基因造血干细胞移植（AHSCT）治疗是MF患者唯一的治愈选择。然而，由于发病率和死亡率较高，AHSCT受到严格限制。Janus激酶2（JAK2）抑制剂鲁索替尼是伴有脾肿大和全身症状的中危-II或高危MF患者的一线治疗药物，但大多数MF患者会对鲁索替尼产生耐药或不耐受。因此，MF治疗是医学界面临的一项挑战。本综述总结了MF治疗的3个研究方向：JAK抑制剂单药治疗、非JAK靶向药物单药治疗、鲁索替尼与其他药物联合治疗。我们强调鲁索替尼与其他药物联合是一种有前景的策略。
