About this trial
This study is a multicenter, prospective, single-arm exploratory clinical trial designed to evaluate the efficacy and safety of prothrombin complex concentrate (PCC) in the treatment of bleeding episodes in patients with hemophilia A with inhibitors. All participants received on-demand PCC therapy during bleeding episodes, with dosing adjusted by investigators according to the type of bleeding. The recommended dose was 50 IU/kg per infusion, administered every 8-12 hours, with a maximum total daily dose not exceeding 150 IU/kg. If no effective hemostasis was achieved within 24 hours, investigators could decide to add other hemostatic agents or switch to alternative treatments.
Eligibility criteria
Qualifiers
Diagnosed with hemophilia A with inhibitors. For high-responding patients (those who have previously had an inhibitor titer >5 BU), the inhibitor titer at enrollment must be >0.6 BU;
Age between 12 and 65 years;
At least three joint bleeding episodes within the past six months;
Signed informed consent form.
Disqualifiers
Presence of other congenital or acquired bleeding disorders;
Liver function tests (ALT, AST) >2.5 times the upper limit of normal, or renal function tests (BUN, Cr) >1.5 times the upper limit of normal;
Currently receiving immune tolerance induction (ITI) therapy with an inhibitor titer <5 BU;
History of thrombotic events;
Trial design
Treatments tested in this trial
- PCC
Treatment groups
Sponsors and collaborators
Institute of Hematology & Blood Diseases Hospital, China
Lead sponsor
North China University of Science and Technology
Collaborator
The Second Affiliated Hospital of Kunming Medical University
Collaborator
The Second People's Hospital of Anhui Province
Collaborator
Children's Hospital of Chongqing Medical University
Collaborator
Union Hospital, Affiliated to Fujian Medical University
Collaborator
The First Affiliated Hospital of Xiamen University
Collaborator
First Affiliated Hospital of Guangxi Medical University
Collaborator