Copernicus Memorial Hospital, Lodz, Poland.
Department of Experimental Hematology, Medical University of Lodz, Lodz, Poland.
Eur J Haematol. 2019 Dec;103(6):531-541. doi: 10.1111/ejh.13319. Epub 2019 Sep 18.
Immune thrombocytopenia (ITP) is an autoimmune disease characterized by platelet destruction and reduced platelet production resulting in decreased platelet level and an increased risk of bleeding. Based on the immunologic mechanism of ITP, front-line standard therapy consists of corticosteroids and intravenous immunoglobulins (IVIG). If patients do not respond to the first-line treatment, or if continuous therapy is required, the disorder is called refractory ITP, and second-line therapy is indicated. This treatment may consist of rituximab, thrombopoietin receptor agonists, splenectomy, or cytotoxic drugs. Despite significant advances, many patients do not respond to any the treatments listed below, and new treatment options need to be developed for this relapsed and refractory group. Recent clinical studies have indicated promising outcomes for novel drugs, either as single agents or in combination with traditional drugs. This review discusses the latest and the most promising novel drugs for ITP in adults.
免疫性血小板减少症 (ITP) 是一种自身免疫性疾病，其特征为血小板破坏和减少血小板生成，导致血小板计数降低和出血风险增加。根据 ITP 的免疫机制，一线标准治疗包括皮质类固醇和静脉注射免疫球蛋白 (IVIG)。如果患者对一线治疗无反应，或者需要持续治疗，则该疾病称为难治性 ITP，需要二线治疗。这种治疗可能包括利妥昔单抗、血小板生成素受体激动剂、脾切除术或细胞毒性药物。尽管取得了重大进展，但许多患者对以下列出的任何治疗都没有反应，需要为这一复发和难治性群体开发新的治疗选择。最近的临床研究表明，新型药物作为单一药物或与传统药物联合使用，具有有前景的疗效。本文讨论了成人 ITP 的最新和最有前途的新型药物。
