Salter Brittany M, Crowther Mark Andrew
Department of Medicine, McMaster University, Hamilton, Ontario, Canada.
Hematology Am Soc Hematol Educ Program. 2024 Dec 6;2024(1):214-221. doi: 10.1182/hematology.2024000544.
Catastrophic antiphospholipid syndrome (CAPS) is a rare but life-threatening form of antiphospholipid syndrome (APS) defined by the rapid onset of large and small vessel thrombosis occurring simultaneously across multiple sites, resulting in multiorgan dysfunction. The presence of underlying immune dysfunction causing activation of coagulation and, in many cases, abnormal complement regulation predisposes these patients to thrombotic events. CAPS is often preceded by triggering factors such as infection, surgery, trauma, anticoagulation discontinuation, and malignancy. Given the high mortality rate, which may exceed 50%, prompt recognition and initiation of management is required. The detection of antiphospholipid antibodies and the histopathologic identification of microvascular ischemia via tissue biopsy are required to diagnose CAPS. However, these patients are often too unwell to obtain results and wait for them. As such, investigations should not delay CAPS therapy, especially if there is strong clinical suspicion. Management of CAPS requires "triple therapy" with glucocorticoids, intravenous heparin, therapeutic plasma exchange, and/or intravenous immunoglobulin. Treatment for refractory disease is based on poor-quality evidence but includes anti-CD20 (rituximab) or anticomplement (eculizumab) monoclonal antibodies and other immunosuppressant agents, either alone or in combination. The rarity of this syndrome and the subsequent lack of randomized clinical trials have led to a paucity of high-quality evidence to guide management. Continued international collaboration to expand ongoing CAPS registries will allow a better understanding of the response to newer targeted therapy.
灾难性抗磷脂综合征（CAPS）是抗磷脂综合征（APS）的一种罕见但危及生命的形式，其定义为多个部位同时迅速发生大、小血管血栓形成，导致多器官功能障碍。潜在的免疫功能障碍导致凝血激活，在许多情况下，补体调节异常使这些患者易发生血栓事件。CAPS通常由感染、手术、创伤、抗凝药物停用和恶性肿瘤等触发因素诱发。鉴于其死亡率可能超过50%，需要迅速识别并开始治疗。诊断CAPS需要检测抗磷脂抗体以及通过组织活检进行微血管缺血的组织病理学鉴定。然而，这些患者往往病情太重，无法获得结果并等待结果。因此，检查不应延误CAPS治疗，特别是在临床高度怀疑的情况下。CAPS的治疗需要糖皮质激素、静脉注射肝素、治疗性血浆置换和/或静脉注射免疫球蛋白进行“三联疗法”。难治性疾病的治疗基于质量较差的证据，但包括抗CD20（利妥昔单抗）或抗补体（依库珠单抗）单克隆抗体以及其他免疫抑制剂，可单独使用或联合使用。这种综合征的罕见性以及随后缺乏随机临床试验导致缺乏高质量的证据来指导治疗。持续的国际合作以扩大正在进行的CAPS登记，将有助于更好地了解对更新的靶向治疗的反应。
