hemophilia A
Conditions
Interventions
research group (Part 3) :Model guidance
control group (Part 3) :no
Observational Studies (Part 2) :no
Questionnaire Survey (Part 1) children with hemophilia:no
Sponsors
Beijing Children’s Hospital, Capital Medical University
Eligibility
Sex/Gender
All
Age
4 Years to 18 Years
Inclusion criteria
Inclusion criteria: part 1 hemophilia A or B (FVIII or FIX=40IU/dL); Age: 4-18 years old. part 2 ? a clear diagnosis of haemophilia major or intermediate (FVIII or FIX:C = 5IU/dL). (ii) Age 4-16 years at enrolment (ensure that the last follow-up visit has been completed at this unit). (iii) Have received prophylactic treatment including concentrated human/recombinant clotting factors for more than six months prior to enrolment. (iv) Cumulative exposure days of more than 50 days; negative inhibitor at entry (<0.6 BU/ml, confirmed by 2 independent tests). ? regular clinical visits to our centre and availability of visit data. (vi) PK testing has been performed and the child's own individual PK parameters are available. ? Written informed consent from the child's legal guardian can be obtained prior to enrolment. part 3 ? a clear diagnosis of haemophilia major or intermediate (FVIII or FIX:C = 5IU/dL). (ii) Age 4-16 years at enrolment (ensure that the last follow-up visit has been completed at this unit). (iii) Have received prophylactic treatment including concentrated human/recombinant clotting factors for more than six months prior to enrolment. (iv) Cumulative exposure days of more than 50 days; negative inhibitor at entry (<0.6 BU/ml, confirmed by 2 independent tests). ? regular clinical visits to our centre and availability of visit data. (vi) PK testing has been performed and the child's own individual PK parameters are available. ? Written informed consent from the child's legal guardian can be obtained prior to enrolment.
Exclusion criteria
Exclusion criteria: Children with a combination of other bleeding system disorders that the investigator considers unsuitable for enrolment. Detection of FVIII/FIX inhibitor (=0.6 BU/ml, confirmed by 2 independent tests).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Sports participation rate;Types of sports; | — |
Secondary
| Measure | Time frame |
|---|---|
| Magnetic resonance (IPSG) of the target joint;score of ultrasound;Hemophilia Joint Health Score;Annual bleeding rate;Annualized rate of joint bleeding;Annualized spontaneous bleeding rate;Zero bleeding ratio;Zero joint bleeding ratio;Zero spontaneous bleeding rate;life quality score;physical profile;FVIII Annual consumption and number of venipunctures; | — |
Countries
China
Contacts
Public ContactRunhui Wu
Beijing Children’s Hospital, Capital Medical University
Outcome results
None listed