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.
Non-interventional study: subjects to be selected according to the usual clinical practice of their physician
Study Type
OBSERVATIONAL
Enrollment
80
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.
Allgemeines Krankenhaus Linz, Kinderklinik
Linz, Austria
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
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Sonnengesundheitszentrum
München, Bavaria, Germany
Werlhof-Institut für Haemostaseologie GmbH
Hanover, Lower Saxony, Germany
Institut für Thrombophilie und Hämostaseologie
Münster, North Rhine-Westphalia, Germany
Vivantes Klinikum im Friedrichshain
Berlin, Germany
Charite Campus Virchow-Klinikum, Padiatrie mit S. Hamatologie und Onkologie
Berlin, Germany
Kinder- und Jugendarzt-Praxis Blaubeuren
Blaubeuren Abbey, Germany
Institute of Experimental Haematology and Transfusion Medicine
Bonn, Germany
Praxis fur Kinder- und Jugendmedizin, Homoopathie
Brannenburg, Germany
Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik
Bremen, Germany
...and 11 more locations
Time frame: 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.
Time frame: 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.
Time frame: End of study visit (any time up to 8.7 years)
Mean Total Number of Bleeding Episodes in Participants
Participants documented all bleeding episodes in a diary during the study.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Baseline up to 8.7 years