Department of Leukemia, The University of Texas Md Anderson Cancer Center, Houston, Texas, USA.
Expert Rev Hematol. 2022 Mar;15(3):233-241. doi: 10.1080/17474086.2022.2057296. Epub 2022 Mar 29.
Given the progressive nature of myelofibrosis, the incidence of thrombocytopenia increases over time. Furthermore, approved drugs ruxolitinib and fedratinib, induce thrombocytopenia. Hence, treatment of myelofibrosis patients with low platelet counts is an unmet need.
This review summarizes the current and emerging treatment options available for patients with myelofibrosis and thrombocytopenia. In the first section of this review, we summarized the use of JAK inhibitors in patients with thrombocytopenia, and in the second part, we focused on use of therapies other than JAK Inhibitors such as steroids, immunomodulatory agents, androgens and other novel agents.
Up to 25% of patients with myelofibrosis have platelet counts below 100,000 at presentation. Patients with thrombocytopenia are more likely to be anemic and PRBC transfusion-dependent, as well as have high-risk disease characteristics and a poor overall survival rate. Among all JAK inhibitors studied in phase 3 clinical trials, pacritinib seems not to induce significant thrombocytopenia while maintaining a good spleen response. Severe thrombocytopenia is a major impediment to myelofibrosis therapy, and more research, particularly on novel therapeutic agents aimed at cytopenic patient populations, is needed.
鉴于骨髓纤维化的进展性，血小板减少症的发病率随时间推移而增加。此外，已批准的药物鲁索替尼和 fedratinib 会引起血小板减少症。因此，治疗血小板计数低的骨髓纤维化患者是未满足的需求。
这篇综述总结了目前和新兴的治疗骨髓纤维化伴血小板减少症患者的治疗选择。在这篇综述的第一部分，我们总结了 JAK 抑制剂在血小板减少症患者中的应用，在第二部分，我们重点介绍了 JAK 抑制剂以外的治疗方法，如类固醇、免疫调节剂、雄激素和其他新型药物。
多达 25%的骨髓纤维化患者在就诊时血小板计数低于 100,000。血小板减少症患者更容易贫血和依赖红细胞输血，并且具有高危疾病特征和较差的总体生存率。在所有在 3 期临床试验中研究的 JAK 抑制剂中，pacritinib 似乎不会引起明显的血小板减少症，同时保持良好的脾脏反应。严重的血小板减少症是骨髓纤维化治疗的主要障碍，需要进行更多的研究，特别是针对针对细胞减少症患者群体的新型治疗药物。
