PCC Treatment for Hemophilia Patients With Inhibitor(2022PCC-A)

Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
Age12-65
SponsorInstitute of Hematology & Blood Diseases Hospital, China

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

30 Participants
are divided into 1 treatment group

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