Hemophilia A With Inhibitor
Conditions
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.
Interventions
DRUGRituximab
375mg/Square meter for consecutive 4 months
Domastic plasma derived factor FVIII (with intermidiate vWF) 50IU/kg every other day
DRUGPrednisone
2mg/kg every day for 4 weeks then typering in 3 months
Sponsors
Beijing Children's Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
1 Years to 14 Years
Healthy volunteers
No
Inclusion criteria
* Males * from 1 to 14 years old * severe or moderate hemophilia A; * inhibitors positive before ITI started.
Exclusion criteria
* Females * \<1 or \>14 years old * hemophilia B or mild haemophilia A; * inhibitor negative before ITI started.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Success rate | 2 years | Success rate |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Annualized Bleeding Rate | 2 year | How many times for all types of bleeding |
| Annualized Joint Bleeding Rate | 2 year | How many times for joint bleeding |
| Success time | 2 years | How long to success |
Countries
China
Contacts
Primary ContactXin Ni, master
Outcome results
None listed