Congenital Bleeding Disorder, Haemophilia A, Haemophilia A With Inhibitors, Haemophilia B, Haemophilia B With Inhibitors
Conditions
Brief summary
This study is conducted in Africa and Asia. The aim of this study is to evaluate in the participating countries the orthopaedic status and the degree of arthropathy of severe haemophilia patients in general.
Interventions
OTHERNo treatment given
Subject will only fill out a questionaire
Sponsors
Novo Nordisk A/S
Study design
Observational model
ECOLOGIC_OR_COMMUNITY
Time perspective
CROSS_SECTIONAL
Eligibility
Sex/Gender
MALE
Age
6 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* Patient (and/or parents or the patient's legally acceptable representative, if applicable) must give signed and dated informed consent before enrolment in the study * Male patients at least 6 years old with diagnosis of severe congenital haemophilia A or B with or without inhibitors * Patients receiving on demand replacement factors/bypassing agents therapy
Exclusion criteria
* Clinically relevant coagulation disorders other than congenital haemophilia A or B * Patients on currently active treatment for HCV (Hepatitis C Virus) or HIV (Human Immune Deficiency Virus) infections
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Type of haemophilia and inhibitor characteristics: Against FVIII or FIX; high or low titre; anamnestic response (high or low responder) | After 6 months (recruitment and data collection) |
| Clinical (using Haemophilia joint score) and radiological (using Pettersson score) orthopaedic status of defined joints: Elbow, knees and ankles in relation to haemophilia A or B | After 6 months (recruitment and data collection) |
Secondary
| Measure | Time frame |
|---|---|
| Quality of Life - EQ-5D (Euro Quality - 5 Domains) questionnaire | After 6 months (recruitment and data collection) |
| Mean orthopaedic score in the 4 groups according to the Pettersson and Haemophilia joint scores | After 6 months (recruitment and data collection) |
| Living characteristics of the patient's household | After 6 months (recruitment and data collection) |
| Economic aspects of the management of haemophiliacs and its burden on patient/family and community resources | After 6 months (recruitment and data collection) |
| Usage of anti haemophilic treatment in IU/kg | During the last year preceding patient recruitment |
Countries
India, Morocco, Oman, South Africa
Outcome results
None listed