Conditions: Hemophilia A
Keywords: Acquired Hemophilia A Anti-Factor VIII antibodies Anti-Factor VIII inhibitors Hemophilia A Factor VIII inhibitors
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 2
Healthy volunteers: yes

Brief summary:
The purpose of this study is to evaluate the rate of response when administering rituximab to suppress or eliminate the anti-body in a patient's blood that inhibits the effectiveness of their factor replacement product compared to treatment using cyclophosphamide. This is a Phase 2/3 study to find out what effects (good and bad) and response rituximab has on a patient and their anti-Factor VIII antibodies. Also, to compare the effect (good and bad) of the rituximab with cyclophosphamide on a patient and their anti-Factor VIII antibodies to see which is better. This research is being done because we do not know which treatment regimen (rituximab or cyclophosphamide) is more effective in eliminating or suppressing the anti-Factor VIII antibody in patients with acquired Hemophilia A.

Detailed description:
This is a prospective Phase II randomized multi-institutional controlled pilot trial comparing the regimen of single agent rituximab with 6 weeks cytotoxic therapy with oral cyclophosphamide to eradicate or suppress autoimmune anti-factor VIII antibodies in individuals with acquired hemophilia A. Patients will be randomized to receive either of these two regimens when their autoimmune anti-factor VIII antibodies prove to be refractory to initial upfront immunosuppressive treatment with oral prednisone 1 mg/kg/day (or equivalent corticosteroid doses) for 3 weeks. Patients will be randomized to the treatment cohorts according to the biostatistical methods.

Arms:
arm 1: Patients will receive rituximab. arm 2: Patients will receive oral cyclophosphamide.

Interventions: intervention 1: Rituxan intervention 2: prednisone
Intervention descriptions: intervention 1: Acquired Hemophilia A Patients Who Have Developed Anti-Factor VIII Antibodies intervention 2: \<30 mg/day

Protocol document: