Abstract:
Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare hematologic disease caused by autoantibodies against ADAMTS-13 that trigger microangiopathic hemolytic anemia. Therapeutic plasma exchange and glucocorticoids have been the mainstay of treatment for the past 30 years. In 2019, caplacizumab was approved as an addition to this regimen for the acute treatment of iTTP. Randomized controlled trials and real-world evidence have shown that caplacizumab reduces the time to platelet count normalization, refractoriness, and exacerbations of the disease, with an acceptable safety profile. In the past 5 years, there have been arguments against the upfront use of caplacizumab in all patients with iTTP, particularly related to the perceived lack of clinical benefit, safety concerns related to bleeding risk, and high costs. This perspective aimed to address these concerns in the context of the experience of expert centers that have used the drug for >5 years.
摘要:
免疫介导的血栓性血小板减少性紫癜(iTTP)是一种罕见的血液学疾病,由针对ADAMTS13的自身抗体引起,可引发微血管病性溶血性贫血。在过去的30年中,治疗性血浆置换和糖皮质激素一直是治疗的主要手段。2019年,caplacizumab被批准作为iTTP急性治疗方案的补充。随机对照试验和现实世界的证据表明,caplacizumab可以减少血小板计数正常化的时间。折射,和疾病的恶化,具有可接受的安全性。在过去的五年里,有人反对在所有iTTP患者中预先使用caplacizumab,特别是与感知到的缺乏临床益处有关,与出血风险相关的安全问题,和高成本。这种观点旨在根据使用该药物超过五年的专家中心的经验来解决这些问题。