Arslan Davulcu Eren, Demirci Zühal, Yılmaz Umut, Ar Muhlis Cem, Teke Hava Üsküdar, Karakuş Volkan, Çiftçiler Rafiye, Selim Cem, Yavaşoğlu İrfan, Durusoy Salih Sertaç, Okan Vahap, Akdeniz Aydan, Yolcu Alkım, Aydoğdu İsmet, Güney Tekin, Yılmaz Asu Fergün, Şahin Fahri
University of Health Sciences Bakırkoy Dr. Sadi Konuk Training and Research Hospital, Hematology Clinic, Istanbul, Turkey.
Hematology Department, Ege Adult Haemophilia and Thrombosis Center, Ege University Medical Faculty, İzmir, Turkey.
Indian J Hematol Blood Transfus. 2023 Jan;39(1):107-115. doi: 10.1007/s12288-022-01556-8. Epub 2022 Jul 31.
Acquired hemophilia A (AHA) is a rare disease caused by autoantibodies inhibiting factor VIII (FVIII) activity. Although the conditionis usually idiopathic, there may be other underlying diseases. Treatment consists of two steps: treatment of acute bleeding and immunosuppression. In this multicenter study, we aimed to demonstrate the clinical characteristics, management details, and survival of AHA patients in Turkey. Data was collected from eleven centers in Turkey. aPTT, FVIII, FVIII inhibitor, and hemoglobin (HB) levels, mixing test results, and demographics at diagnosis, treatment information, adverse events, bleeding episodes during follow-up, relapses, and outcome were analyzed. Twenty-nine patients were analyzed (58.6% female). No underlying disorder could be detected in 14 patients. The most prevalent etiologies were pregnancy, malignancy and infections. The median FVIII activity and FVIII inhibitor titer at diagnosis were 0.7% (0.0-29.4%) and 32.6 BU (0.6-135.6 BU) respectively. Bleeding was severe in 44.8% of patients. The HB value was significantly lower in patients with severe bleeding. Most of the patients (n = 25, 86.2%) had only one bleeding episode without relapse, three patients (10.3%) had two bleeding episodes, and one patient had more than three bleedings. 21 (75%) patients received hemostatic therapy. The use of recombinant FVIIa was slightly higher than activated prothrombin complex concentrate (15 versus 10 patients). Immunosuppressive treatment was initiated in 26 (93%) patients. Regimens containing steroid, cyclophosphamide, and rituximab in different combinations were the most preferred. The median follow-up period was 13 months (2-156 months). Median overall survival was 154.97 months. Four and six-year survival were 90.9 ± 0.8% and 77.9 ± 14.1% respectively. This is a unique study that investigated the demographic characteristics, treatment approaches, and patient survival of AHA in Turkey.
获得性血友病A（AHA）是一种由抑制凝血因子VIII（FVIII）活性的自身抗体引起的罕见疾病。尽管该病通常为特发性，但可能存在其他潜在疾病。治疗包括两个步骤：急性出血的治疗和免疫抑制。在这项多中心研究中，我们旨在展示土耳其AHA患者的临床特征、管理细节和生存率。数据收集自土耳其的11个中心。分析了活化部分凝血活酶时间（aPTT）、FVIII、FVIII抑制剂和血红蛋白（HB）水平、混合试验结果以及诊断时的人口统计学数据、治疗信息、不良事件、随访期间的出血发作、复发情况和结局。共分析了29例患者（58.6%为女性）。14例患者未检测到潜在疾病。最常见的病因是妊娠、恶性肿瘤和感染。诊断时FVIII活性和FVIII抑制剂滴度的中位数分别为0.7%（0.0 - 29.4%）和32.6 Bethesda单位（BU）（0.6 - 135.6 BU）。44.8%的患者出血严重。严重出血患者的HB值显著更低。大多数患者（n = 25，86.2%）仅有一次出血发作且无复发，3例患者（10.3%）有两次出血发作，1例患者有三次以上出血。21例（75%）患者接受了止血治疗。重组FVIIa的使用略高于活化凝血酶原复合物浓缩剂（分别为15例和10例患者）。26例（93%）患者开始了免疫抑制治疗。最常用的方案是包含类固醇、环磷酰胺和利妥昔单抗的不同组合。中位随访期为13个月（2 - 156个月）。中位总生存期为154.97个月。4年和6年生存率分别为90.9 ± 0.8%和77.9 ± 14.1%。这是一项独特的研究，调查了土耳其AHA患者的人口统计学特征、治疗方法和患者生存率。
