Prophylaxis Versus On-demand Therapy Through Economic Report

Study Overview
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Brief Summary
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The project is a controlled observational, multicenter, prospective data collection on secondary prophylaxis with Kogenate Bayer in adolescents and adults with severe haemophilia A (FVIII < 1%). The observational period will cover at least 5 years per patient. The long-term secondary prophylaxis group will be compared versus on-demand treatment group by the assessment of orthopedic status progression and pharmacoeconomics evaluation.

Official Title
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Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A. POTTER

Conditions
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Hemophilia A

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


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: age ≥ 12 years and ≤ 55 years severe haemophilia A (FVIII < 1%) absence of inhibitors (Bethesda titre < 0.6 BU/ml) Previous Treated Patients (prior exposure days > 200) Kogenate Bayer administered 20-30 IU/kg -3 times a week, for the prophylaxis group ≥ 6 joint bleeds requiring treatment with FVIII concentrates in the previous 6 months before enrollment, for on-demand group written informed consent Exclusion Criteria: concomitant severe and chronic diseases or congenital skeletal malformation unreliability of patient or likelihood of follow-up failure presence of inhibitors or history of inhibitors (in the previous 2 years) currently on immune tolerance treatment hepatic cirrhosis or liver disease in rapid progression AIDS platelet count < 75,000/mm3 presence of conditions that influence negatively patient´s compliance participation in another study

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 55 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> | Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)<br>* Long-term secondary prophylaxis with product administered 20-30 UI /kg three times weekly<br>|
| Group 2<br> | Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)<br>* On-demand treatment with product given only for bleeding episodes<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Evaluate the efficacy, safety and pharmacoeconomic of long-term secondary prophylaxis with Kogenate Bayer in comparison to on-demand treatment with Kogenate Bayer in terms of joint bleeding episodes per year |  | Every 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy, safety, pharmacoeconomic impact of secondary prophylaxis versus on demand group in terms of overall bleeding episodes |  | Every 6 months | 
| Muscolo skeletal evaluation by Orthopedic Joint Score |  | Every 12 months | 
| Radiological Evaluation by Pettersson Score |  | Baseline and after 3-5 years | 
| Health related quality of life |  | Every 12 months | 
| cost-effectiveness and utility, patient compliance, adverse events |  | Every 6 months |