The non factor replacement therapy of emicizumab will be applied to patients with hemophilia with and without inhibitor as prophylaxis. The subcutaneous injection of emicizumab of the loading dose of 3 mg/kg per week for 4 weeks, followed by the maintenance doses ranging from 1.5 mg/kg weekly to 6 mg/kg every 4 weeks is attractive and convenient for clinical practice. Hemophilia quality of life non factor replacement therapy
Conditions
Interventions
Group 1 has 4 adult hemophilia patients with and without inhibitors.,Group 2 has 5 pediatric hemophilia patients with and without inhibitors.
Treatment,Treatment
Group 1,Group 2
Sponsors
None listed
Eligibility
Sex/Gender
Male
Age
12 Years to 70 Years
Inclusion criteria
Inclusion criteria: 1. Patients with moderate to severe hemophilia A (factor VIII activity less than 3%) with or without inhibitor. 2. Age 12 years or older 3. Access to treatment for acute bleeding within 2 hours 4. Experienced low-dose prophylaxis or at least 5 bleeds in 6 months in episodic treatment 5. Written informed consent is obtained from the patients, parents of caregivers
Exclusion criteria
Exclusion criteria: 1. Other coagulation disorders 2. Liver or renal diseases 3. Conditions that may increase risk of bleeding 4. Concomitant disease, condition or abnormality that could interfere with study conduct or pose unacceptable risk to patients 5. Required elective surgery 6. Patients parents or caregivers refuse to participate in the study 7. Patients parents or caregivers withdraw anytime during the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Qulity of life 7 months Haemo-QoL | — |
Secondary
| Measure | Time frame |
|---|---|
| Episode of bleeding 7 months Number | — |
Countries
Thailand
Contacts
Public ContactAmpaiwan Chuansumrit
Faculty of Medicine Ramathibodi Hospital, Mahidol University
Outcome results
None listed