A national French noninterventional study to assess the long-term safety and efficacy of reformulated nonacog alfa
| Autor: | Marc Trossaert, Ségolène Claeyssens-Donadel, Thierry Lambert, Chantal Rothschild, Annie Borel-Derlon, Sepideh Attal, Fabienne Volot |
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| Rok vydání: | 2017 |
| Předmět: |
medicine.medical_specialty
Pediatrics Allergic reaction business.industry Treatment regimen Immunology Hematology 030204 cardiovascular system & hematology 03 medical and health sciences Regimen 0302 clinical medicine Usual care Lack of efficacy Physical therapy Immunology and Allergy Medicine Long term safety business 030215 immunology Recombinant factor IX |
| Zdroj: | Transfusion. 57:1066-1071 |
| ISSN: | 0041-1132 |
| Popis: | BACKGROUND Nonacog alfa, the recombinant Factor IX (F IX) used for the treatment of hemophilia B, was approved in Europe in 1998. A reformulated version was approved for European use in 2007. STUDY DESIGN AND METHODS This postmarketing study, as recommended by the risk management plan, was conducted to confirm the safety of reformulated nonacog alfa in a usual care setting in France. This open-label, noninterventional, prospective, longitudinal postmarketing study comprised 19 French hemophilia centers. Patients with hemophilia B receiving reformulated nonacog alfa for prophylaxis or on-demand treatment were followed up on usual care schedule. RESULTS A total of 58 subjects were enrolled, of whom 29 (50%) were less than 18 years of age. Hemophilia was severe (baseline F IX activity |
| Databáze: | OpenAIRE |
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