Journal article
Results from a large multinational clinical trial (guardian™1) using prophylactic treatment with turoctocog alfa in adolescent and adult patients with severe haemophilia A: safety and efficacy
Haemophilia : the official journal of the World Federation of Hemophilia, Vol.19(5), pp.691-697
09/2013
DOI: 10.1111/hae.12159
PMID: 23647704
Abstract
Summary
Recombinant factor VIII (rFVIII) products provide a safe and efficacious replacement therapy for prophylaxis and treatment of bleeding episodes in patients with severe haemophilia A. This multinational, open‐label, non‐controlled trial investigated the safety and efficacy of turoctocog alfa, a new rFVIII product. The primary objective was to evaluate safety. A total of 150 patients (24 adolescents and 126 adults) with severe haemophilia A (FVIII activity ≤1%), with at least 150 exposure days (EDs) to any FVIII product and no history of inhibitors were enrolled, and 146 patients (97%) completed the trial. All patients received prophylaxis with turoctocog alfa for approximately 6 months and had a mean of 85 EDs during the trial. None of the patients developed FVIII inhibitors, there were no indications of early FVIII inhibitor development and no safety concerns were identified. A total of 225 adverse events were reported in 100 (67%) patients, with the most common being events associated with dosing procedures, headaches, and nasopharyngitis. A total of 499 bleeding episodes were reported during the trial, the majority (89%) were controlled with 1–2 infusions of turoctocog alfa. Based on patient reports, the success rate (defined as ‘excellent’ or ‘good’ haemostatic response) for treatment of bleeding episodes was 81%. The overall median annualized bleeding rate was 3.7 (interquartile range: 8.7) bleeds/patient/year. In conclusion, turoctocog alfa provides a new, safe and effective alternative for prophylaxis and treatment of bleeding episodes in patients with haemophilia A.
Details
- Title: Subtitle
- Results from a large multinational clinical trial (guardian™1) using prophylactic treatment with turoctocog alfa in adolescent and adult patients with severe haemophilia A: safety and efficacy
- Creators
- S. R Lentz - The University of Iowa Carver College of MedicineM Misgav - Tel Aviv UniversityM Ozelo - University of CampinasS. Z Šalek - University Hospital CentreD Veljkovic - Institute for Mother and Child Health Care of SerbiaM Recht - Oregon Health & Science UniversityM Cerqueira - HEMORIOA Tiede - Medizinische Hochschule HannoverB Brand - University HospitalM. E Mancuso - Fondazione IRCCS Ca' Granda Ospedale Maggiore PoliclinicoS Seremetis - Novo Nordisk IncA Lindblom - Novo Nordisk A/SU Martinowitz - Tel Aviv University
- Resource Type
- Journal article
- Publication Details
- Haemophilia : the official journal of the World Federation of Hemophilia, Vol.19(5), pp.691-697
- DOI
- 10.1111/hae.12159
- PMID
- 23647704
- ISSN
- 1351-8216
- eISSN
- 1365-2516
- Number of pages
- 7
- Grant note
- Novo Nordisk A/S
- Language
- English
- Date published
- 09/2013
- Academic Unit
- Hematology, Oncology, and Blood & Marrow Transplantation; Internal Medicine
- Record Identifier
- 9984094323002771
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