Hemophilia and Thrombosis Centre, University Hospital of Bari, Piazza Giulio Cesare 11, 70124 Bari, Italy.
Medicina (Kaunas). 2023 Sep 28;59(10):1739. doi: 10.3390/medicina59101739.
Acquired Hemophilia A (AHA) is a rare autoimmune disorder characterized by the onset of a sudden and unexpected bleeding episode in a patient with no personal or family history of bleeding diathesis, and with a typical laboratory feature, i.e., a prolonged activated partial thromboplastin time that is not otherwise explained. This bleeding disorder is caused by autoantibodies directed against the coagulation factor VIII (FVIII). AHA is idiopathic in 50% of cases and is secondary to well-defined diseases in the remaining 50%. AHA affects elderly patients although it has also been observed in the post-partum period. Bleeding manifestations are heterogeneous, ranging from mild to life-threatening bleeds involving limbs and organs. Severe bleeding with a significant decrease in hemoglobin levels must be promptly and adequately treated in order to avoid a worsening of the hemorrhages and their complications. According to international recommendations, the bypass agents (i.e., activated prothrombin complex concentrate and activated recombinant factor VII) and the replacement therapy with recombinant porcine FVIII are considered as the first-line therapy for bleeding control, due to their proven clinical efficacy. Plasma-derived or recombinant FVIII concentrates could be used as second-line treatments. Emicizumab may represent a valid and interesting therapeutic option for prophylaxis of bleeding recurrences.
获得性血友病 A（AHA）是一种罕见的自身免疫性疾病，其特征是在无出血素质个人或家族史的患者中突然出现意外出血事件，且具有典型的实验室特征，即延长的活化部分凝血活酶时间，且无法用其他原因解释。这种出血性疾病是由针对凝血因子 VIII（FVIII）的自身抗体引起的。50%的 AHA 为特发性，其余 50%则继发于明确的疾病。AHA 可发生于老年患者，但也可见于产后。出血表现呈异质性，从轻度到危及生命的四肢和器官出血均有。严重出血并伴有血红蛋白水平显著下降时，必须及时充分治疗，以避免出血恶化及其并发症。根据国际建议，旁路制剂（即活化的凝血酶原复合物浓缩物和活化的重组因子 VII）和重组猪 FVIII 替代疗法被认为是控制出血的一线治疗方法，因为它们具有已证实的临床疗效。血浆源性或重组 FVIII 浓缩物可用作二线治疗。依库珠单抗可能是预防出血复发的一种有效且有意义的治疗选择。
