State-of-the-Art Review on Myelofibrosis Therapies |
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| Institution: | 1. Beijing Key Laboratory of Resistant Gene Resources and Molecular Development, College of Life Sciences, Beijing Normal University, Beijing, China;2. 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;3. Department of Hematology, Fourth Medical Center, Chinese PLA General Hospital, Beijing, China;1. Department of Medicine, Division of Hematology, The University of North Carolina at Chapel Hill, Chapel Hill, NC;2. Lineberger Comprehensive Cancer Center, The University of North Carolina at Chapel Hill, Chapel Hill, NC;3. Department of Medicine, Division of Hematology-Oncology, University of Rochester, Rochester, NY |
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| Abstract: | 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. |
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