The non-factor therapy of emicizumab, a humanized bispecific monoclonal antibody binding factor IXa and factor X, has shown its effectiveness in preventing bleeding episodes by creating adequate factor Xa to promote clot formation. It can be applied to patients with hemophilia with and without inhibitor as prophylaxis. The subcutaneous injection of emicizumab of the loading dose of 3 mcg/kg per week for 4 weeks, followed by the maintenance doses ranging from 1.5 mcg/kg weekly to 6 mcg/kg every 4
Conditions
Interventions
1) A low dose monthly emicizumab more than 1 mcg/kg by subcutaneous injection will be administered for 12 months. The emicizumab injection must be obtained within 72 hours from the targeted injection
Active Comparator Drug
Group 1
Sponsors
None listed
Eligibility
Sex/Gender
Male
Age
2 Years to 20 Years
Inclusion criteria
Inclusion criteria: 1. Patients with hemophilia A without inhibitor 2. Age 2 to 20 years old under the coverage of the National Health Security Office (NHSO) 3. Written informed consent is obtained from the patients, parents or caregivers
Exclusion criteria
Exclusion criteria: 1. Patients with hemophilia A with inhibitor 2. Patients with hemophilia B 3. Patients hypersensitive to emicizumab
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Annual bleeding rate 12 months record the bleeding episode during the prophylaxis period | — |
Secondary
| Measure | Time frame |
|---|---|
| CHOKLATE 12 months quality of life assessment using questionnaire | — |
Countries
Thailand
Contacts
Public ContactAmpaiwan Chuansumrit
Faculty of Medicine Ramathibodi Hospital, Mahidol University
Outcome results
None listed