Registry For Patients Treated With BeneFix In Usual Care Setting In Germany

Study Overview
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Brief Summary
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The purpose of this observational study is to describe the incidence of adverse events among patients treated with BeneFix® in usual health care settings in Germany.

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 Benefix (Registered) In Germany And Austria

Conditions
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Hemophilia B

Intervention / Treatment
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* Drug: BeneFIX


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with hemophilia B already receiving or starting treatment with reformulated BeneFIX®. Exclusion Criteria: Patients with hemophilia B treated with a product other than BeneFIX®. Inclusion in the ongoing prospective registry of European hemophilia B patients using BeneFIX®.
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 |
| --- | --- |
| A<br>Patients with Hemophilia B | Drug: BeneFIX<br>* Patients will be treated in accordance with the requirements of the labeling of BeneFIX in Germany. The dosage and duration of therapy is to be determined by the physician to meet the patients' individual needs for treatment.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | An AE was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability or incapacity; cancer; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to last visit (up to 8.7 years) that were absent before treatment or that worsened relative to pretreatment state. AEs included both serious and non-serious. | Baseline until last visit (up to 8.7 years) | 
| Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events (SAEs) | Treatment-related AE was any untoward medical occurrence attributed to study drug in a participant who received study drug. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; cancer; congenital anomaly. AEs included both serious and non-serious. Relatedness to BeneFIX was assessed by the investigator. | Baseline until last visit (up to 8.7 years) | 
| Number of Participants With Factor IX (FIX) Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay | FIX inhibitor development was defined as measured inhibitor titer of greater than (>) 0.6 Bethesda Units (BU) using the Nijmegen-modified Bethesda assay. | Baseline until last visit (up to 8.7 years) | 
| Number of Participants With Adverse Events (AEs) of Special Interest | An AE was any untoward medical occurrence in a participant who received study treatment without regard to possibility of causal relationship. Adverse Events of special interest included allergic reactions, less than expected therapeutic effect (LETE) of drug, lack of efficacy/low recovery, erythrocyte agglutination in tube or syringe red blood cell (RBC) agglutination phenomena and thrombogenicity. | Baseline until last visit (up to 8.7 years) | 
| Investigator Assessment of Treatment Tolerability of Participants | Investigator assessed the tolerability of participants and categorized as very good, good, moderate and poor. | End of study visit (any time up to 8.7 years) | 
| Participant Assessment of Treatment Tolerability | Participants evaluated their treatment (BeneFIX) tolerability and rated it in 4 categories as very good, good, moderate and poor. | End of study visit (any time up to 8.7 years) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean Total Number of Bleeding Episodes in Participants | Participants documented all bleeding episodes in a diary during the study. | Baseline until last visit (up to 8.7 years) | 
| Mean Total Number of Bleeding Episodes Per Year in Participants | Participants documented all bleeding episodes in a diary during the study. Mean total number of bleeding episodes per year was calculated by mean total number of bleeding episodes divided by duration of observation period (in years) for bleeding documentation. | Baseline until last visit (up to 8.7 years) | 
| Number of Participants With Change From Baseline Status in Number of Days Missed From School or Work | Change from baseline status in days missed from school or work was categorized in 3 categories: Improvement, unchanged and worsening. Improvement was defined as a decrease in number of days missed by participants from school/work as compared to baseline; worsening was defined as an increase in number of days missed by participants from school/work as compared to baseline; unchanged was defined as no change in number of days missed by participants from school/work as compared to baseline. In this outcome measure, number of participants with change from baseline status (as improved, worsen, unchanged) in days missed from school/work were reported. | Baseline, up to 8.7 years | 
| Investigator Assessment of Treatment Efficacy of Participants | Investigator evaluated the efficacy of BeneFIX in participants and rated it in 4 categories as very good, good, moderate and poor. | End of study visit (any time up to 8.7 years) | 
| Investigator Assessment of Treatment Handling of Participants | Investigator evaluated the handling (administration) of BeneFIX by participants and rated it in 4 categories as very good, good, moderate and poor. | End of study visit (any time up to 8.7 years) | 
| Assessment of Treatment Efficacy by the Participants | Participants evaluated the efficacy of BeneFIX and rated it in 4 categories as very good, good, moderate and poor. | End of study visit (any time up to 8.7 years) | 
| Assessment of Treatment Handling by the Participants | Participants evaluated the handling (administration) of BeneFIX and rated it in 4 categories as very good, good, moderate and poor. | End of study visit (any time up to 8.7 years) | 
| Investigator Assessment of Treatment Satisfaction of Participants | Investigator evaluated the participant's satisfaction of treatment with BeneFIX and rated it in 4 categories as very satisfied, satisfied, unsatisfied and very unsatisfied. | Baseline up to 8.7 years |