Study Evaluating Refacto For Pharmacovigilance

Study Overview
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Brief Summary
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The purpose of this study is to investigate the effectiveness and safety of treatment with ReFacto under conditions of routine therapy. Furthermore a continuous benefit/risk assessment will be done.

Detailed Description
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Non-interventional study: subjects to be selected according to the usual clinical practice of their physician

Official Title
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Pharmacovigilance Evaluation Of Refacto In Usual Care Settings

Conditions
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Hemophilia A

Intervention / Treatment
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* Drug: Moroctocog alfa


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Proven diagnosis of Hemophilia A Exclusion Criteria: Contraindications according to Summary of Product Characteristics
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 |
| --- | --- |
| A<br>Patients with Hemophilia A | Drug: Moroctocog alfa<br>* Patients will be treated in accordance with the requirements of the labeling of ReFacto (Moroctocog alfa) in Germany. The dosage and duration of therapy is to be determined by the physician to meet the patients' individual needs for treatment.<br>* Other names: ReFacto;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean Number of Bleeding Episodes Per Patient Year | Participants with hemophilia A suffer from a hereditary lack of blood clotting factor VIII. As a consequence, the ability of the blood to coagulate is reduced and bleedings at any site or organ of the body may occur after minor injury or even spontaneously. Predominantly, joints, muscles, and internal organs are affected by bleeding complications. Participants reported the occurrence of each bleeding episode while on study. The bleeding rate for each participant was calculated by number of reported episodes per years on study. | Baseline up to a mean duration of 54 months | 
| Mean Number of Bleeding-related Exposure Days Per Patient Year | Exposure days are the number of days of treatment with ReFacto. | Baseline up to a mean duration of 54 months | 
| Mean Number of Exposure Days Per Patient Year | Exposure days are the number of days of treatment with ReFacto. | Baseline up to a mean duration of 54 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Non-serious Adverse Events (AEs) and Serious Adverse Events (SAEs) | AEs are any undesired side effect which occurred in a participant undergoing study treatment independent of whether a correlation with study treatment was suspected or not. SAEs are undesired events which were lethal or life-threatening, made hospitalization or extension of hospital stay necessary, lead to permanent damage with handicap (inability to work), as well as congenital anomalies, malignant disease, or overdosing. Also presence of inhibitors, thrombotic events, erythrocyte agglutination, allergic reactions, less than therapeutic effect, and inhibitor development were considered SAEs. | Baseline up to a mean duration of 54 months | 
| Number of Participants With de Novo Inhibitor Formation | The applied criteria of clinical relevance for de novo inhibitor formation was defined as normal Factor VIII dosage was ineffective to control a bleeding, control of bleeding episodes required increasing Factor VIII dosage, change of concentrate type (administration of activated Prothrombin-Complex Concentrate [aPCC] or recombinant Factor VII [rFVII ]) was needed to stop a bleeding, or change of therapy strategy (intensive prophylaxis or Immune Tolerance Induction [ITI]) was required. | Baseline up to a mean duration of 54 months | 
| Mean Annual ReFacto Consumption Per Patient Year | ReFacto administered as International Units (IU) according to the physician's decision following the drug's summary of product characteristics (SPC) and according to usual care principles. | Baseline up to a mean duration of 54 months | 
| Number of Participants for Physicians' Assessment of Satisfaction With Treatment Success | Subjective assessment by the physician to evaluate treatment success (i.e., control of bleeding, Factor VIII consumption, treatment efficacy and tolerance, handling of preparation, and days missing from work or school). Physician rated assessment could be categorized as Very satisfied, Satisfied, Unsatisfied, or Very unsatisfied; no criteria was pre-specified for the assessment categories in this observational study. | Baseline up to a mean duration of 54 months | 
| Number of Participants for Physicians' Assessment of Efficacy | Subjective assessment by the physician to evaluate control of bleeding. Physician rated assessment could be categorized as Very good, Good, Moderate, or Poor; no criteria was pre-specified for the assessment categories in this observational study. | Baseline up to a mean duration of 54 months | 
| Number of Participants for Patients' Assessment of Efficacy | Subjective assessment by the participant to evaluate control of bleeding. Patient rated assessment could be categorized as Very good, Good, Moderate, Poor, or No specification; no criteria was pre-specified for the assessment categories in this observational study. | Baseline up to a mean duration of 54 months | 
| Number of Participants for Physicians' Assessment of Tolerance | Subjective assessment by the physician to evaluate the participants' tolerance of treatment with ReFacto (i.e., dose, administration method, or adverse effects). Physician rated assessment could be categorized as Very good, Good, Moderate, or Poor; no criteria was pre-specified for the assessment categories in this observational study. | Baseline up to a mean duration of 54 months | 
| Number of Participants for Patients' Assessment of Tolerance | Subjective assessment by the participant to evaluate tolerance of treatment with ReFacto (i.e., dose, administration method, or adverse effects). Patient rated assessment could be categorized as Very good, Good, Moderate, Poor, or No specification; no criteria was pre-specified for the assessment categories in this observational study. | Baseline up to a mean duration of 54 months | 
| Number of Participants for Physicians' Assessment of Handling of ReFacto | Subjective assessment by the physician to evaluate the participants' handling (preparation and administration) of ReFacto. Physician rated assessment could be categorized as Very good, Good, Moderate, or Poor; no criteria was pre-specified for the assessment categories in this observational study. | Baseline up to a mean duration of 54 months | 
| Number of Participants for Patients' Assessment of Handling of ReFacto | Subjective assessment by the participant on handling (preparation and administration) of ReFacto. Patient rated assessment could be categorized as Very good, Good, Moderate, Poor, or No specification; no criteria was pre-specified for the assessment categories in this observational study. | Baseline up to a mean duration of 54 months | 
| Number of Participants for Days of Sick Leave Per Month | Days of sick leave (missing work or school) per month categorized as No days of absence, Number of days of absence, Long-term inability to work or study, Not employed or at school, or No specification. | Baseline up to a mean duration of 54 months | 

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