Beijing Children's Hospital, Capital Medical University
Trial Snapshot
- Phase
- Phase 4
- Sponsor
- Enrollment
- 55
- Locations
- 1
- Primary Endpoint
- Success rate
Study Overview
Brief Summary
The study start on January 18, 2017. The Severe(FⅧ<1%) and moderate hemophilia A (FⅧ1%～5%)children with high titer inhibitor(historical peak inhibitor titer≥5BU ) combining with poor ITI risk(s) were enrolled. The low-dose ITI was alone or combined with immunosuppression.
Detailed Description
Poor risk(s) includes:①peak historical inhibitor titer≥200BU ②inhibitor titer≥10BU before ITI initiation ③peak inhibitor titer during ITI≥200BU ④time to titer decline to<10BU before ITI≥24 months ⑤age≥8 years at start of ITI ⑥ITI initiated ≥5 years after inhibitor diagnosis ⑦interruptions in ITI≥2 weeks in duration. The low-dose ITI strategy consist of FⅧ（25-50IU/kg）alone or combining with immunosuppression: prednisone and Rituximab when the inhibitor titer ≥40BU ml/ml before or during ITI.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 1 Year to 14 Years (Child)
- Sex
- Male
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •from 1 to 14 years old
- •severe or moderate hemophilia A;
- •inhibitors positive before ITI started.
Exclusion Criteria
- •<1 or >14 years old
- •hemophilia B or mild haemophilia A;
- •inhibitor negative before ITI started.
Arms & Interventions
ITI strategy
The low-dose ITI was Coagulation Factor VIII (50IU/kg, every other day) alone or combined with prednisone (2mg Kg-1/day, one month, then taper in three months) depending on the tendency of inhibitor, and Rituximab (375mg/square meter every week for 4 weeks) when the inhibitor titer ≥40BU/ml before or during ITI.Inhibitor and hemorrhage should be retested and recorded periodically.
Intervention: Coagulation Factor VIII (Drug)
ITI strategy
The low-dose ITI was Coagulation Factor VIII (50IU/kg, every other day) alone or combined with prednisone (2mg Kg-1/day, one month, then taper in three months) depending on the tendency of inhibitor, and Rituximab (375mg/square meter every week for 4 weeks) when the inhibitor titer ≥40BU/ml before or during ITI.Inhibitor and hemorrhage should be retested and recorded periodically.
Intervention: Prednisone (Drug)
ITI strategy
The low-dose ITI was Coagulation Factor VIII (50IU/kg, every other day) alone or combined with prednisone (2mg Kg-1/day, one month, then taper in three months) depending on the tendency of inhibitor, and Rituximab (375mg/square meter every week for 4 weeks) when the inhibitor titer ≥40BU/ml before or during ITI.Inhibitor and hemorrhage should be retested and recorded periodically.
Intervention: Rituximab (Drug)
Outcomes
Primary Outcomes
Success rate
Time Frame: 2 years
Success rate
Secondary Outcomes
- Annualized Bleeding Rate(2 year)
- Success time(2 years)
- Annualized Joint Bleeding Rate(2 year)
Investigators
Runhui WU
Capital Medical Univercity
Beijing Children's Hospital