Abstract：
Acquired thrombotic thrombocytopenic purpura (aTTP) is a thrombotic microangiopathy resulting in high mortality. Caplacizumab is approved for treatment of adults with acquired thrombotic thrombocytopenic purpura that has shown faster platelet normalization, clinical improvement, and reduced risk of recurrent/refractory disease. We report 2 cases of adolescents treated off-label with caplacizumab who were able to stop before 30 days from end of plasma exchange after platelets normalized and ADAMTS13 activity recovered to >20% to 30%. Our results show similar efficacy to other reports of patients under 18 receiving caplacizumab in first line, regardless of plasma exchange strategy, and may offer insight into early cessation criteria.
摘要：
获得性血栓性血小板减少性紫癜（aTTP）是一种导致高死亡率的血栓性微血管病。Caplacizumab被批准用于治疗患有获得性血栓性血小板减少性紫癜的成人，其显示出更快的血小板正常化。临床改善，降低复发/难治性疾病的风险。我们报告了2例接受caplacizumab治疗的青少年患者，他们能够在血小板正常化和ADAMTS13活性恢复至>20％至30％后，在血浆置换结束后30天内停止治疗。我们的结果显示，与其他18岁以下患者一线接受caplacizumab的疗效相似，无论血浆置换策略如何，并可能提供有关早期戒烟标准的见解。
