Hemophilia
Conditions
Keywords
Hemophilia, Turkey, Observational, Adult
Brief summary
The project is an observational, multi-central, prospective, non-interventional and open-label data collection study on secondary prophylaxis with recombinant FVIII products in adolescents and adults with severe hemophilia A (FVIII \< 1%). It will be a controlled observation of patients on secondary prophylaxis versus on-demand treatment regimen. Patients will be enrolled preferably on a 1:1 basis with regards to prophylaxis and on-demand treatment. The patient enrollment period will be 2 years with a follow-up (observation period) of 2 years for each patient. Based on the primary effectiveness parameters (joint bleeds and overall bleeds per year) an observation period of 2 years is considered sufficient although it has to be admitted that it is rather short to assess the progression of orthopedic status. Previously treated prophylaxis patients with at least 50 exposure days and patients with continuing prophylaxis treatment will be included.
Interventions
OTHERRecombinant Factor VIII (Kogenate, BAY14-2222)
All dosage, frequency and duration for drugs will be under the decision of the treating physician.
Sponsors
Bayer
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Age ≥ 18 years * Severe hemophilia A (FVIII\<1%) diagnosis * Prior treatment or ongoing treatment with on-demand or prophylaxis regimens according to Turkish guidelines and requirements * Previously treated patients with at least \>50 exposure days * Written informed consent signed by patient/legal representative
Exclusion criteria
* Currently on immune tolerance treatment * Platelet count \< 75,000/mm3 * Participation in another study * Existence of inhibitors in the past and in the last currently available blood sample prior to study start (Bethesda titer \< 0.6 BU/ml) * Existence of inhibitor history in family members who also are diagnosed with hemophilia A * Having been on primary prophylaxis as defined in the introduction * Participation in another study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Median ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group | After 4 years |
| Mean ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group | After 4 years |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Musculoskeletal evaluation recommended by World Federation of Hemophilia: Radiological evaluation (Pettersson Score) | After 4 years | — |
| Cost-effectiveness (cost of additional joint bleed) | After 4 years | — |
| Cost-utility | After 4 years | The costs of care of subjects with haemophilia will be evaluated adopting the perspective of the payer, i.e. the Social Security Institution \[SSI\]. |
| Number of overall bleeding episodes | After 4 years | — |
| Number of spontaneous bleeds | After 4 years | — |
| Quality of life as measured with the SF-36 | Baseline and after 4 years | — |
| Quality of life as measured with Hemo-QoL | Baseline and after 4 years | — |
| Comparison of patient compliance between prophylaxis and on-demand therapy groups | After 4 years | — |
| Musculoskeletal evaluation recommended by World Federation of Hemophilia: Orthopedic Joint Score (Gilbert Score) | After 4 years | — |
Countries
Turkey (Türkiye)
Outcome results
None listed