Division of Hematology/Oncology, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA; Department of Leukemia, University of Texas MD Anderson Cancer Center, 1400 Holcombe Blvd, FC4.3039, Houston, TX, 77030, USA.
Adult Leukemia Program, Division of Hematology/Oncology, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, One Gustave L Levy Place, Box 1079, New York, NY 10029, USA; Myeloproliferative Disorders Clinical Research Program, Division of Hematology/Oncology, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, One Gustave L Levy Place, Box 1079, New York, NY 10029, USA.
Hematol Oncol Clin North Am. 2021 Apr;35(2):353-373. doi: 10.1016/j.hoc.2020.12.003. Epub 2021 Jan 27.
The US Food and Drug Administration (FDA) approval of Janus kinase 2 inhibitors, ruxolitinib and fedratinib for the treatment of intermediate-2 or high-risk primary or secondary myelofibrosis (MF) has revolutionized the management of MF. Nevertheless, these drugs do not reliably alter the natural history of disease. Burgeoning understanding of the molecular pathogenesis and the bone marrow microenvironment in MF has galvanized the development of targeted therapeutics. This review provides insight into the novel therapies under clinical evaluation.
美国食品和药物管理局 (FDA) 批准使用 Janus 激酶 2 抑制剂芦可替尼和 fedratinib 治疗中危-2 或高危原发性或继发性骨髓纤维化 (MF),这彻底改变了 MF 的治疗模式。然而,这些药物并不能可靠地改变疾病的自然病程。人们对 MF 中的分子发病机制和骨髓微环境的认识不断加深,推动了靶向治疗的发展。本文对正在进行临床评估的新型疗法进行了综述。