Lemiale Virginie, Valade Sandrine, Mariotte Eric
Medical Intensive Care Unit, Saint Louis University Hospital, Assistance Publique des Hôpitaux de Paris, Paris, France.
Ther Clin Risk Manag. 2021 Jun 3;17:577-587. doi: 10.2147/TCRM.S205632. eCollection 2021.
Thrombotic thrombocytopenic purpura (TTP) is a thrombotic microangiopathy secondary to a severely decreased A Disintegrin And Metalloprotease with ThromboSpondin type 1 repeats 13 (ADAMTS13) activity, resulting in the formation of widespread von Willebrand factor - and platelet-rich microthrombi. ADAMTS13 deficiency is mainly acquired through anti-ADAMTS13 autoantibodies in adults. With modern standards of care, unresponsive TTP has become rarer with a frequency of refractory/relapsing forms dropping from >40% to <10%. As patients with unresponsive TTP are at increased risk of mortality, prompt recognition and early therapeutic intensification are mandatory. Therapeutic options at the disposal of clinicians caring for patients with refractory TTP consist of increased ADAMTS13 supplementation, increased immunosuppression, and inhibition of von Willebrand factor adhesion to platelets. In this work, we focus on possible therapies for the management of patients with unresponsive TTP, and propose an algorithm for the management of these difficult cases.
血栓性血小板减少性紫癜（TTP）是一种血栓性微血管病，继发于具有1型血小板反应蛋白基序的解聚素样金属蛋白酶13（ADAMTS13）活性严重降低，导致广泛形成富含血管性血友病因子和血小板的微血栓。ADAMTS13缺乏在成人中主要是通过抗ADAMTS13自身抗体获得。按照现代治疗标准，难治性TTP已变得更为罕见，难治性/复发性形式的发生率从>40%降至<10%。由于难治性TTP患者的死亡风险增加，必须迅速识别并尽早加强治疗。负责治疗难治性TTP患者的临床医生可采用的治疗选择包括增加ADAMTS13补充、增加免疫抑制以及抑制血管性血友病因子与血小板的黏附。在这项工作中，我们专注于难治性TTP患者管理的可能疗法，并提出了一种针对这些疑难病例的管理算法。
