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Myelofibrosis (MF) is a myeloproliferative neoplasms (MPN), usually accomplies with thrombocytopenia, severely impacts the life quality of patients and presents a risk for leukemic transformation. Pacritinib is a kind of Janus activated kinase 2 (JAK2) and Fms-like tyrosine kinase 3 (FLT3) inhibitor. U.S. FDA has granted accelerated approval for pacritinib capsules (Vonjo), on Feb 28, 2022, in the treatment of adult patients with the platelet (blood clotting cells) levels below 50 000·μL-1 who have a rare form of bone marrow disorder known as intermediate or high-risk primary and secondary myelofibrosis. The oral dose of 200 mg twice daily showed good clinical efficacy and manageable safety. In this paper, the mechanism of action, pharmacodynamics, pharmacokinetics, safety, and clinical efficacy of daridorexant are reviewed.
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骨髓纤维化(MF)是一种骨髓增殖性肿瘤,常伴有血小板减少症,严重影响患者的生活质量并存在向白血病转化的风险。帕克替尼是一种Janus活化激酶2(JAK2)及Fms样酪氨酸激酶3(FLT3)抑制剂。帕克替尼胶囊(商品名:Vonjo)于2022年2月28日被美国FDA加速批准,用于治疗患有罕见形式的骨髓疾病(中危或高危的原发性或继发性MF)且血小板(凝血细胞)水平低于50 000·μL-1的成人患者,口服200 mg,bid的剂量显示出良好的临床疗效和可控的安全性。本文对其作用机制、药动学、药效学、临床疗效及安全性进行综述,为临床应用提供参考。
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