Apheresis & Cellular Therapy Unit, Department of Hemotherapy and Hemostasis, ICMHO, IDIBAPS, Hospital Clínic, University of Barcelona, Barcelona Catalonia, Spain.
Apheresis & Cellular Therapy Unit, Department of Hemotherapy and Hemostasis, ICMHO, IDIBAPS, Hospital Clínic, University of Barcelona, Barcelona Catalonia, Spain.
Transfus Apher Sci. 2021 Feb;60(1):103011. doi: 10.1016/j.transci.2020.103011. Epub 2020 Nov 7.
Autoimmune thrombotic thrombocytopenic purpura (aTTP) is a severe disease caused by the production of autoantibodies against von Willebrand factor (vWF)-cleaving ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin-1 motifs; 13th member of the family). In 2018, caplacizumab was approved for the treatment of patients with acute aTTP in conjunction with plasma exchange (PE) and immunosuppressive therapy. Immunosuppressive standard of care includes mainly steroids whereas rituximab is usually reserved for refractory cases. We report three patients with a first acute episode of aTTP who were successfully treated with a paradigm-changing scheme including standard of care (caplacizumab, steroids and PE) plus upfront therapy with rituximab and intravenous immunoglobulins (CASPERI). Rituximab was added 1-4 days after diagnosis, when ADAMTS13 autoantibodies were detected and intravenous immunoglobulins were administered after performing PE using albumin as replacement solution. Successful outcome was observed in all three patients: platelet recovery (>150 × 10/L) was observed after 3, 4, and 5 days from diagnosis; ADAMTS13 activity >5% and ADAMTS13 autoantibodies were negative after 14, 15, and 21 days from diagnosis. In conclusion, caplacizumab, steroids, PE (using fresh frozen plasma or albumin as replacement solution and adding intravenous immunoglobulins) plus upfront rituximab therapy was a safe and efficient combination to induce remission in case of acute aTTP.
自身免疫性血栓性血小板减少性紫癜（aTTP）是一种由抗血管性血友病因子（vWF）裂解 ADAMTS13（一种具有血小板反应蛋白-1 基序的解整合素金属蛋白酶；家族的第 13 个成员）的自身抗体引起的严重疾病。2018 年，卡普雷珠单抗被批准与血浆置换（PE）和免疫抑制疗法联合用于治疗急性 aTTP 患者。免疫抑制的标准治疗包括主要使用类固醇，而利妥昔单抗通常保留用于难治性病例。我们报告了 3 例首次急性 aTTP 患者，他们成功地接受了一种改变治疗模式的方案治疗，该方案包括标准治疗（卡普雷珠单抗、类固醇和 PE）加利妥昔单抗和静脉注射免疫球蛋白（CASPERI）的起始治疗。在诊断后 1-4 天添加利妥昔单抗，当检测到 ADAMTS13 自身抗体并在用白蛋白作为替代溶液进行 PE 后，给予静脉注射免疫球蛋白。所有 3 例患者均获得成功：从诊断开始后 3、4 和 5 天观察到血小板恢复（>150×10/L）；从诊断开始后 14、15 和 21 天 ADAMTS13 活性>5%和 ADAMTS13 自身抗体阴性。总之，卡普雷珠单抗、类固醇、PE（使用新鲜冷冻血浆或白蛋白作为替代溶液，并添加静脉注射免疫球蛋白）加利妥昔单抗起始治疗是一种安全有效的联合治疗方法，可诱导急性 aTTP 缓解。
