Rodriguez-Merchan E Carlos
Department of Orthopedic Surgery, La Paz University Hospital-IdiPaz, Madrid, Spain.
Expert Rev Hematol. 2023 Jul-Dec;16(9):651-658. doi: 10.1080/17474086.2023.2232547. Epub 2023 Jul 4.
It is important to know the current status of hemophilic arthropathy diagnoses, treatments, complications, and outcomes in developed countries.
A bibliographic search in PubMed for articles published from 1 January 2019 through 12 June 2023 was performed.
In developed countries with specialized hemophilia treatment centers, primary hematological prophylaxis (started before the age of 2 years and after no more than one joint bleed) has almost completely eliminated the joint-related problems of the disease. The ideal goal of zero hemarthroses can be achieved only with intense and well-dosed prophylaxis: intravenous infusion of coagulation factor - standard half-life or extended half-life; periodic or subcutaneous injections of nonfactor products (emicizumab or fitusiran). However, hemophilic arthropathy continues to occur due to subclinical joint hemorrhages. In one study, 16% of the joints without reported hemarthroses showed signs of previous subclinical bleeding (hemosiderin deposits with/without synovial hypertrophy on magnetic resonance imaging were deemed signs of previous subclinical bleeding), rendering evidence for subclinical bleeding in people with severe hemophilia with lifelong prophylaxis treatment. Subclinical joint hemorrhages can be averted only by employing accurate and tailored prophylaxis.
了解发达国家血友病性关节病的诊断、治疗、并发症及预后的现状很重要。
对2019年1月1日至2023年6月12日期间发表在PubMed上的文章进行了文献检索。
在设有血友病专科治疗中心的发达国家,一级血液学预防(在2岁之前且在不超过一次关节出血后开始)几乎完全消除了该疾病与关节相关的问题。只有通过强化且剂量合适的预防措施才能实现零关节积血的理想目标:静脉输注凝血因子——标准半衰期或延长半衰期的;定期或皮下注射非因子产品(依美珠单抗或fitusiran)。然而,由于亚临床关节出血,血友病性关节病仍会发生。在一项研究中,16%未报告有关节积血的关节显示出先前亚临床出血的迹象(磁共振成像上有/无滑膜肥大的含铁血黄素沉积被视为先前亚临床出血的迹象),这为接受终身预防治疗的重度血友病患者存在亚临床出血提供了证据。只有采用准确且量身定制的预防措施才能避免亚临床关节出血。