hemophilia A
Conditions
Interventions
research group:Use drug concentration to guide treatment
Sponsors
Beijing Children’s Hospital, Capital Medical University
Eligibility
Sex/Gender
Male
Age
1 Years to 18 Years
Inclusion criteria
Inclusion criteria: 1.A clear diagnosis of moderate/severe hemophilia A (FVIII<=2IU/dL); 2.The age at enrollment is <18 years old; 3.Bispecific antibody (emecizumab) is being used or is planned to be used for prophylaxis. 4. Regular clinical visits to the center and the data of the visits are available; 5. The children can obtain the written informed consent of their legal guardians before enrolling in the group.
Exclusion criteria
Exclusion criteria: 1. Patients complicated with other bleeding system diseases and considered by the investigator as unsuitable for enrollment; 2. Patients with thrombotic disease or family history of related diseases. 3. Patients with a previous allergy to monoclonal antibody therapy, emicizumab, or the presence of allergic disease.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| annual bleeding rate;joint function;joint structure;life quality scores; | — |
Countries
China
Contacts
Public ContactZhenping Chen
Outcome results
None listed