hemophilia
Conditions
Interventions
No joint disease group:Preventive treatment program
Relevant segment lesion group:Individualized trough concentration target values were determined according to joint lesions, and prevention and treatment plans were formulated according to PK
Sponsors
Beijing Children's Hospital
Eligibility
Sex/Gender
Male
Age
1 Years to 14 Years
Inclusion criteria
Inclusion criteria: 1.Severe hemophilia A(F VIII: C<1%); 2.Aged 1 to 14 years; 3.Preventive treatment was started at the time of enrollment (regular use of coagulation factors before bleeding to prevent bleeding); 4.The use of concentrated human source/recombinant coagulation factor VIII has accumulated more than 50 days of exposure; 5.There were negative inhibitors at the time of enrollment, and there was no inhibitor-positive history and family history; 6.Clinical visits are available on a regular basis and data is available, and preventive treatment is available prior to enrollment; 7.Children can obtain the informed consent of their legal guardians before enrollment.
Exclusion criteria
Exclusion criteria: 1.Children with other hemorrhagic diseases or other systemic diseases that the researcher considers unsuitable for inclusion; 2.F VIII inhibitor was found (>=0.6BU, confirmed by 2 separate tests); 3.Unable to comply with the study protocol or refusing to continue to participate in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| trough concentration level;Joint lesion; | — |
Secondary
| Measure | Time frame |
|---|---|
| Imaging evaluation of joint;Bleeding control index;Activity level indicator;Life quality indicator;Cost; | — |
Countries
China
Contacts
Public ContactWu Runhui
Beijing Children's Hospital
Outcome results
None listed