First report on the safety and efficacy of an extended half-life glycoPEGylated recombinant FVIII for major surgery in severe haemophilia A
| Autor: | Kingsley Hampton, Pratima Chowdary, Lotte Jacobsen, Elena Santagostino, Hideyuki Takedani, Silke Ehrenforth, Jameela Sathar, Clifford M. Takemoto, Claude Negrier, Scott Dunkley, Anne T. Neff |
|---|---|
| Rok vydání: | 2017 |
| Předmět: |
Adult
Male medicine.medical_specialty Adolescent Joint replacement medicine.medical_treatment Haemophilia A Synovectomy Hemorrhage 030204 cardiovascular system & hematology Hemophilia A Recombinant factor viii Severity of Illness Index Polyethylene Glycols 03 medical and health sciences Young Adult 0302 clinical medicine Outcome Assessment Health Care Clinical endpoint medicine Humans Genetics (clinical) Aged Factor VIII business.industry Hematology General Medicine Turoctocog alfa Length of Stay Middle Aged medicine.disease Interim analysis Surgery Surgical Procedures Operative Severe haemophilia A business 030215 immunology Half-Life |
| Zdroj: | Haemophilia : the official journal of the World Federation of Hemophilia. 23(5) |
| ISSN: | 1365-2516 |
| Popis: | Background N8-GP (turoctocog alfa pegol) is an extended half-life glycoPEGylated recombinant factor VIII (FVIII) product developed for the prevention and treatment of bleeds in haemophilia A patients. Aim This is a planned interim analysis of pathfinder™3, an international, open-label, Phase 3 trial evaluating the efficacy and safety (including immunogenicity) of N8-GP administered before, during and after major surgery in severe haemophilia A patients aged ≥12 years. Methods Sixteen patients who underwent 18 major surgical procedures (including synovectomy, joint replacement and ankle arthrodesis) were included here. Postoperative assessments were conducted daily for days 1–6, and once for days 7–14. Primary endpoint was N8-GP haemostatic efficacy, assessed after completion of surgery using a four-point scale (‘excellent’, ‘good’, ‘moderate’, ‘none’). Results Haemostasis was successful (rated ‘excellent’ or ‘good’) on completion of surgery in 17 (94.4%) procedures and rated as ‘moderate’ (5.6%) for one surgery in a patient with multiple comorbidities who needed an intraoperative N8-GP dose (20.7 IU kg−1). In the postoperative period, three bleeds occurred (one during days 1–6; two during days 7–14); all were successfully treated with N8-GP. Mean N8-GP consumption on day of surgery was 80.0 IU kg−1; patients received a mean of 1.7 doses (median: 2, range: 1–3). No safety concerns were identified. Conclusion The data showed that N8-GP was effective and well tolerated for the prevention and treatment of bleeds during major surgery; such FVIII products with extended half-lives may modify current treatment schedules, enabling fewer infusions and earlier patient discharge. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|
Plný text