Cooper Nichola
Hammersmith Hospital, Imperial College, London, UK.
Br J Haematol. 2017 Apr;177(1):39-54. doi: 10.1111/bjh.14515. Epub 2017 Mar 10.
The management of patients with immune thrombocytopenia (ITP) is rapidly evolving. Over the last 15 years, a number of novel treatments have improved practice, with many steroid-sparing agents and a reduction in the progression to splenectomy. Although this has improved clinical care, many therapeutic challenges remain. There is no diagnostic test, no biomarkers to direct treatment and few comparative studies to help management decisions. Development of up to date guidelines is difficult with little high-grade evidence. First line treatment continues to be steroids and intravenous immunoglobulins (IVIG) although both are often poorly tolerated and not curative. Common second line treatments include rituximab, immunosuppressive agents, such as azathioprine and mycophenolate mofetil, and the thrombopoietin receptor agonists romiplostim and eltrombopag. There are no comparative studies to decide between these agents and treatment is generally individualized, depending on comorbidity. Use of splenectomy has declined and is generally reserved for patients with chronic disease, although the exact position of splenectomy is subject to debate. Further understanding of the cause of disease in individual patients may help guide treatment. Randomized controlled studies of common treatments and novel treatments for refractory patients are urgently needed.
免疫性血小板减少症（ITP）患者的管理正在迅速发展。在过去15年中，一些新的治疗方法改善了治疗实践，出现了许多类固醇替代药物，脾切除术的进展也有所减少。尽管这改善了临床护理，但许多治疗挑战仍然存在。没有诊断测试，没有指导治疗的生物标志物，也几乎没有比较研究来帮助做出管理决策。由于缺乏高级别证据，制定最新指南很困难。一线治疗仍然是类固醇和静脉注射免疫球蛋白（IVIG），尽管两者通常耐受性差且无法治愈。常见的二线治疗包括利妥昔单抗、免疫抑制剂，如硫唑嘌呤和霉酚酸酯，以及血小板生成素受体激动剂罗米司亭和艾曲泊帕。目前尚无比较研究来在这些药物之间做出选择，治疗通常根据合并症进行个体化。脾切除术的使用已经减少，一般仅用于慢性病患者，尽管脾切除术的确切地位仍存在争议。进一步了解个体患者的病因可能有助于指导治疗。迫切需要对难治性患者的常用治疗方法和新治疗方法进行随机对照研究。
