Extension at 10 Years of the: Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A.

Study Overview
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Brief Summary
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Following the performance of the POTTER observational study, whose primary objective was to collect data on the benefits of secondary prophylaxis versus on demand treatment in terms of prevention of bleeding episodes, the present study aims to extend the observation time up to 10 years, of the same population involved in the previous study. The present study has been designed to allow a focus on long term disease-related damage at joint level in subjects with severe haemophilia A, as well as on the pharmacoeconomics impact of the two different treatment regimens.

Official Title
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Extension at 10 Years of the: Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A. POTTER-10

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: Completion of follow-up in the original Potter study without having being excluded from the Intention To Treat efficacy analysis of the primary endpoint; Written informed consent specifically issued for the 5-year extension. Exclusion Criteria: Switching of treatment to a Factor VIII concentrate different from Kogenate Bayer/Helixate NexGen after the end of the previous follow-up period.

Ages Eligible for Study
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Minimum Age: 17 Years
Maximum Age: 60 Years

Sexes Eligible for Study
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Male

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 |
| --- | --- |
| Prophylaxis<br>Prophylaxis group: treatment is based on regularly repeated infusions of clotting factor, 20-30 IU/kg -3 times a week | Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)<br>* Administered 20-30 IU/kg 3 times a week<br>|
| On-demand<br>On-demand group: treatment administered when bleeding episode occur | Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)<br>* Administered only for bleeding episodes<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of joint bleeding episodes per year |  | 5 years period | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Total number of bleeding episodes per year |  | 5 years period | 
| Total amount of Recombinant Factor VIII consumption |  | 5 years period | 
| Changes of prophylaxis dose |  | 5 years period | 
| Changes of frequency of infusions |  | 5 years period | 
| Musculoskeletal evaluation by calculating the World Federation of Haemophilia Orthopedic Joint Score (Gilbert score) |  | 5 years period | 
| Proportions of patients who will change the therapeutic regimen during the study |  | 5 years period | 
| Musculoskeletal evaluation by calculating the Radiological score (Pettersson Score) |  | 5 years period | 
| Evaluating the Questionnaires of Health-Related Quality of Life _Haemo Quality of Life (HaemoQol) |  | 5 years period | 
| Evaluating the Questionnaires of Health-Related Quality of Life _Short Form 36 questions (SF36) |  | 5 years period | 
| Evaluating the Questionnaires of Health-Related Quality of Life _ Euro Quality 5 Dimension (EQ5D) |  | 5 years period | 
| Number of days missed from work or school of patients and caregivers because of all the events haemophilia-related |  | 5 years period | 
| Number of patients with poor compliance |  | 5 years period | 
| Number of patients with adverse events |  | 5 years period | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hemophilia A, Congenital