This is a single-center, prospective, single-arm, exploratory study designed to evaluate the efficacy and safety of Bometase Alfa for the on-demand treatment of bleeding episodes in patients with acquired hemophilia A. A total of 20 patients with acquired hemophilia A experiencing bleeding events will be enrolled. Bometase Alfa will be administered at 0.1 U/kg for non-severe bleeding and 0.16 U/kg for severe bleeding, with consecutive doses given at 4-hour intervals until hemostasis is achieved. Treatment will be discontinued once hemostasis is achieved or if symptoms suggestive of arterial thrombosis occur, followed by a safety assessment. Rescue therapy will be initiated if bleeding remains uncontrolled after three consecutive administrations for a single bleeding episode, or if bleeding continues to worsen during treatment and the investigator determines that further treatment with Bometase Alfa is unlikely to provide clinical benefit and may pose a medical risk.
Age ≥18 years;
Isolated prolongation of activated partial thromboplastin time (APTT) with a normal prothrombin time (PT);
Reduced factor VIII coagulant activity (FVIII:C <50%);
Positive FVIII inhibitor, defined as ≥0.6 BU/mL as measured by the Bethesda assay or Nijmegen-modified Bethesda assay, or failure of a 1:1 mixing study with normal plasma to achieve complete correction;
Congenital hemophilia A or B, or any other confirmed congenital coagulation factor deficiency;
Isolated prolonged activated partial thromboplastin time (APTT) due to lupus anticoagulant or antiphospholipid syndrome, with a negative FVIII inhibitor and normal FVIII activity; or coagulation abnormalities caused by disseminated intravascular coagulation (DIC) or severe liver disease that do not fulfill the diagnostic criteria for acquired hemophilia A;
A history or symptoms of any arterial or venous thromboembolic event within 3 months before enrollment, including atherosclerosis, myocardial infarction, ischemic stroke, transient ischemic attack, deep vein thrombosis, or pulmonary embolism, or the presence of DIC;
Use of factor VII (FVII), activated factor VII (FVIIa), tranexamic acid, or aminocaproic acid within 1 day before the planned administration of the study drug; or use of prothrombin complex concentrate (PCC) or factor VIII (FVIII) within 3 days before the first administration;
Single group
Bometase Alfa
DrugFor non-severe bleeding, Bometase Alfa will be administered at a dose of 0.1 U/kg, while patients with severe bleeding will receive 0.16 U/kg. The study drug will be administered consecutively at 4-hour intervals until hemostasis is achieved. Treatment will be discontinued once hemostasis is achieved or if symptoms suggestive of arterial thrombosis occur, after which the patient will enter the safety assessment process.
Incidence of effective hemostasis rate at 8 hours after the first administration
Incidence of effective hemostasis rate at 12 hours after the first administration
Time to achieve clinical hemostasis
Amount of blood product use
Dose of Bometase Alfa administered
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