Factor Product Utilization and Health Outcomes in Patients With Hemophilia
Hemophilia ACongenital2 moreRecombinant factor VIII Fc (rFVIIIFc) and recombinant factor IX Fc (rFIXFc) are extended half-life coagulation factors approved by Health Canada in 2014 for the treatment of severe hemophilia A and B, respectively. The objectives of this observational study is to describe the change in annual factor consumption, clinical and patient-reported outcomes for patients who switch from recombinant factor VIII (rFVIII) and recombinant factor IX (rFIX) to rFVIIIFc/ rFIXFc in Canada, and to explore clinicians' and patients' reasons for switching or not switching.
A Study in Children, Teenagers and Adults With Severe Hemophilia A Who Switched From Other Factor...
Hemophilia AThe main aims of the study are to assess the safety profile of Adynovate as well as how well people respond to the preventive treatment with Adynovate. This study is about reviewing and collecting data of the participants before and after the switch to Adynovate that are already available. No new information will be collected during this study. The total time for data collection in the study will be approximately 72 months (36 months before and 36 months after switching to Adynovate). Participants will not receive Adynovate as part of this study. As participants are not treated in this study, they do not need to visit their doctor in addition to their normal visits.
ATHN 11: Liver Transplantation Outcomes Study
Hemophilia A and BLiver TransplantationThis study is designed as a multi-center, observational cohort study of participants with hemophilia A and B who have and have not undergone liver transplantation. Participants will be asked to complete health related quality of life questionnaires and provide medical history.
Observational Registry of NovoSeven® Used as On-demand Treatment of Bleeds in Patients With Haemophilia...
Congenital Bleeding DisorderHaemophilia A With Inhibitors1 moreThis study was conducted in Africa, Europe, the Middle-East and South America. The primary objective of this registry was to observe the use of single dose and multi-dose use of activated recombinant human factor VII and to compare short-term outcomes, including effectiveness, safety, quality of life and treatment satisfaction with the approved treatments.
Post-marketing Safety Surveillance of NovoSeven® in Patients With Haemophilia and Inhibitors by...
Congenital Bleeding DisorderHaemophilia A1 moreThis study is conducted in Europe. The purpose of this retrospective study is to collect additional safety information of patients with haemophilia and inhibitors who are treated with rFVIIa.
Observational Study on the Efficacy and Safety of NovoSeven® During "Real-life" Usage in Germany...
Congenital Bleeding DisorderCongenital FVII Deficiency3 moreThis NON INTERVENTIONAL OBSERVATIONAL STUDY is conducted in Europe. The primary aim is to observe the haemostatic efficacy of NovoSeven® treatment during routine practice in German clinics. The observational study observes patients with congenital haemophilia with inhibitors to coagulation factors VIII or IX, acquired haemophilia, congenital FVII deficiency, or Glanzmann's thrombasthenia who have received at least one dose of NovoSeven® for treatment of a bleeding episode or for the prevention of a bleeding when undergoing surgery or an invasive procedure.
LTFU for Gene Transfer Subjects With Hemophilia B
Hemophilia BSeveral subjects enrolled in a multi-site, gene transfer clinical study to evaluate the intrahepatic administration of AAV2-hFIX16 vector for the treatment of severe hemophilia B between 2001 and 2009. As the US FDA has established guidelines for the long-term follow-up (LTFU) of subjects receiving investigational gene therapy products, this protocol seeks to characterize the clinical outcome and the type and seriousness of adverse events following the AAV gene transfer. The primary study tools will consist of annual history/physical examination and blood tests, as well as periodic liver ultrasound, to characterize clinical outcomes. Where possible, data will be obtained for up to 15 years following hepatic AAV2-hFIX16 gene transfer.
Study Evaluating Allergic Reactions To Benefix In Hemophilia B Patients
Hemophilia BBased on the finding that anaphylactic reactions to Benefix ("FIX") are associated with a specific IgE, a Basophil histamine release assay was selected to evaluate and demonstrate subject sensitization to FIX.
rFVIIIFc (Elocta®) ITI Chart Review in Patients With Haemophilia A
Hemophilia A With InhibitorA chart review study of patients with haemophilia A with inhibitors treated with rFVIIIFc (Elocta®) for immune tolerance induction.
Safety and Efficacy in a ReAl-Life Study in Patients With Haemophilia Treated wIth NovoEight® for...
Hemophiliathe aim of the SERAPHINE study is to gather data stemming from the French research database BERHLINGO (= Base d'Etude et de Recherche en Hémostase pour Les Investigateurs du Grand-Ouest, i.e. Database for Research on Hemostasis for the Investigators of Western France), in order to get a detailed report about the therapeutic management and use of turoctocog alfa (NovoEight®) in surgery in pwHA (Patient with inherited Hemophilia A).