Effectiveness of Caplacizumab Nanobody in Acquired Thrombotic Thrombocytopenic Purpura Refractory to Conventional Treatment
Effectiveness of Caplacizumab Nanobody in Acquired Thrombotic Thrombocytopenic Purpura Refractory to Conventional Treatment
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Caplacizumab 纳米抗体对常规治疗难治性获得性血栓性血小板减少性紫癜的疗效
DOI：
10.1159/000517813
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发表时间：
2021
影响因子：
2.4
通讯作者：
J. L. Poveda Andrés
中科院分区：
文献类型：
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作者：
T. Palanques;J. Megías;V. Bosó Ribelles;Inés Gómez Seguí;J. L. Poveda Andrés
获得性血小板减少性血栓性紫癜（aTTP）是一种自身抗体介导的疾病，针对具有血栓形成蛋白-1型基序13的酶A去整合素和金属蛋白酶结构域，迄今为止一直采用血浆置换（PEX）和皮质类固醇治疗。一名29岁女性患者在妊娠期间出现aTTP，在接受PEX、皮质类固醇和利妥昔单抗治疗后出现多次复发。最近，caplacizumab，一种抗血管性血友病因子的纳米抗体，已被批准用于治疗aTTP。在我们的患者中，caplacizumab实现了更好的疾病控制，与标准治疗相比，血小板计数恢复时间、PEX天数和住院时间更短，重现了临床试验的结果。Caplacizumab代表了aTTP治疗的重大进展，尤其是在复发性疾病的情况下。
Acquired thrombocytopenic thrombotic purpura (aTTP) is an autoantibody-mediated disease against the enzyme A Disintegrin and Metalloprotease domain with ThromboSpondin-1 type motif 13, which until now has been treated with plasma exchange (PEX) and corticosteroids. A 29-year-old female patient, who presented with aTTP in the context of pregnancy, has developed multiple relapses after treatment with PEX, corticosteroids, and rituximab. Recently, caplacizumab, a nanobody against von Willebrand factor, has been approved for the treatment of aTTP. In our patient, caplacizumab achieved better disease control, with a lower platelet count restoration time, days of PEX and hospitalization duration, as compared to standard therapy, reproducing the results of clinical trials. Caplacizumab represents a significant advance in the treatment of aTTP, especially in cases of recurrent relapses.