Blickstein Dorit
Thrombosis and Hemostasis Unit, Institute of Hematology and Bone Marrow Transplantation, Rambam Health Care Center, Haifa.
Harefuah. 2019 Mar;158(3):196-199.
Primary immune thrombocytopenic purpura (ITP) is an acquired immune-mediated disorder characterized by isolated thrombocytopenia (platelet count less than 100X109/L), caused by IgG autoantibodies which bind to platelets and megakaryocyte, T cell-mediated platelet destruction and impaired megakaryocytic function. Symptoms can manifest as petechiae, purpura, mucosal bleeding and rarely fatal intracranial hemorrhage, as well as reduced quality of life. A wide range of bleeding manifestations exists and it is impossible to tell who will bleed, when and where. The goal of treatment is to prevent severe/life-threatening bleeding. Treatment modalities target various aspects of ITP pathophysiology such as the inhibition of autoantibody production (decreased autoimmune process), modulation of T cell activity (with prolongation of platelets survival), and stimulation of platelet production. The American Society of Hematology and the International Society of Thrombosis and Hemostasis published guidelines on the treatment of ITP patients, where first line treatment focuses on inhibition of autoantibody production and platelet degradation, second-line treatments include immunosuppressive drugs and splenectomy, and third-line treatments aim to stimulate platelet production by megakaryocytes. New available strategies might change the order of treatment lines. As in other situations, treatment should be tailored according to the patient's age, life style, comorbidities and compliance.
原发性免疫性血小板减少症(ITP)是一种获得性免疫介导的疾病,其特征为孤立性血小板减少(血小板计数低于100×10⁹/L),由与血小板和巨核细胞结合的IgG自身抗体、T细胞介导的血小板破坏以及巨核细胞功能受损引起。症状可表现为瘀点、紫癜、黏膜出血,极少出现致命性颅内出血,还会导致生活质量下降。存在广泛的出血表现,无法判断谁会出血、何时何地出血。治疗目标是预防严重/危及生命的出血。治疗方式针对ITP病理生理学的各个方面,如抑制自身抗体产生(减少自身免疫过程)、调节T细胞活性(延长血小板存活时间)以及刺激血小板生成。美国血液学会和国际血栓与止血学会发布了ITP患者的治疗指南,其中一线治疗侧重于抑制自身抗体产生和血小板降解,二线治疗包括免疫抑制药物和脾切除术,三线治疗旨在刺激巨核细胞生成血小板。新的可用策略可能会改变治疗线的顺序。与其他情况一样,治疗应根据患者的年龄、生活方式、合并症和依从性进行调整。