Stasi Roberto, Provan Drew
Department of Medical Sciences, "Regina Apostolorum" Hospital, Albano Laziale, Italy.
Mayo Clin Proc. 2004 Apr;79(4):504-22. doi: 10.4065/79.4.504.
Primary immune thrombocytopenic purpura (ITP), also referred to as idiopathic thrombocytopenic purpura, is an organ-specific autoimmune disorder in which antibody-coated or immune complex-coated platelets are destroyed prematurely by the reticuloendothelial system, resulting in peripheral blood thrombocytopenia. The disease is heterogeneous with regard to its severity and clinical course and is unpredictable in its response to therapy. Although the basic underlying pathophysiology of ITP has been known for more than 50 years, current treatment guidelines are based on expert opinion rather than on evidence because of a lack of high-quality clinical trials and research. The only patients for whom treatment is clearly required are those with severe bleeding and/or extremely low platelet counts (< 10 x 10(9)/L). Treatment of patients with ITP refractory to corticosteroids and splenectomy requires careful evaluation of disease severity, patient characteristics related to risk of bleeding, and adverse effects associated with treatment. Clinical trials with numerous new agents are under way, which we hope will add more effective and targeted strategies to our therapeutic armamentarium. We describe a logical and structured approach to the clinical management of ITP in adults, based on a literature review and our personal experience.
原发性免疫性血小板减少症（ITP），也称为特发性血小板减少性紫癜，是一种器官特异性自身免疫性疾病，其中抗体包被或免疫复合物包被的血小板被网状内皮系统过早破坏，导致外周血血小板减少。该疾病在严重程度和临床病程方面具有异质性，对治疗的反应也不可预测。尽管ITP的基本潜在病理生理学已为人所知50多年，但由于缺乏高质量的临床试验和研究，目前的治疗指南基于专家意见而非证据。明确需要治疗的唯一患者是那些有严重出血和/或血小板计数极低（<10×10⁹/L）的患者。对于对皮质类固醇和脾切除术难治的ITP患者，治疗需要仔细评估疾病严重程度、与出血风险相关的患者特征以及与治疗相关的不良反应。众多新药的临床试验正在进行中，我们希望这将为我们的治疗手段增添更有效和有针对性的策略。我们基于文献综述和个人经验，描述了一种针对成人ITP临床管理的逻辑和结构化方法。
