Comparison of Efficacy, Safety and Costs of Recombinant FVIII Products Between On-demand and Secondary Prophylaxis Groups in Haemophilia A Patients

Study Overview
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Brief Summary
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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.

Official Title
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Evaluation of Efficacy, Safety and Costs of Recombinant FVIII Products Applied to Severe Hemophilia A Patients: Observational Data Collection Study Evaluating On-demand Treatment and Secondary Prophylaxis

Conditions
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Hemophilia

Intervention / Treatment
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* Other: Recombinant Factor VIII (Kogenate, BAY14-2222)


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Group 1<br> | Other: Recombinant Factor VIII (Kogenate, BAY14-2222)<br>* All dosage, frequency and duration for drugs will be under the decision of the treating physician.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | 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 Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of overall bleeding episodes |  | After 4 years | 
| Musculoskeletal evaluation recommended by World Federation of Hemophilia: Orthopedic Joint Score (Gilbert Score) |  | After 4 years | 
| 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 | The costs of care of subjects with haemophilia will be evaluated adopting the perspective of the payer, i.e. the Social Security Institution [SSI]. | After 4 years | 
| Comparison of patient compliance between prophylaxis and on-demand therapy groups |  | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hemophilia, Turkey, Observational, Adult