Canadian clinical experience on switching from standard half-life recombinant factor VIII (rFVIII), octocog alfa, to extended half-life rFVIII, damoctocog alfa pegol, in persons with haemophilia A ≥ 12 years followed in a Comprehensive Hemophilia Care Program in Canada.

INTRODUCTION: Damoctocog alfa pegol (BAY 94-9027, Jivi®) is an extended half-life recombinant factor (F)VIII replacement, indicated for the treatment of haemophilia A in patients aged ≥12 years. Following introduction of damoctocog alfa pegol in Canada in 2020, there have been no reports on routine clinical effectiveness and satisfaction, when switching from a previous FVIII product in Canada. AIM: To report changes in pharmacokinetics, effectiveness, utilization and patient satisfaction when switching to damoctocog alfa pegol prophylaxis from previous standard half-life octocog alfa (BAY 81-8973, Kovaltry®) treatment. METHODS: A single-centre, intra-patient comparison of pharmacokinetics and clinical outcomes was performed. Blood samples drawn once pre-dose and ≥2 times post-dose were measured by a one-stage assay to assess pharmacokinetic parameters including area under the curve (AUC, primary endpoint). Patient-reported outcomes data were collected using the Patient-Reported Outcomes, Burdens and Experiences questionnaire (PROBE). Clinical outcomes included annualized bleeding rate (ABR) and factor utilization. RESULTS: Dose-normalized AUC was significantly increased after switch to damoctocog alfa pegol from octocog alfa. Median (quartile [Q]1; Q3) annualized bleeding rates were 0.67 (0.00; 1.33) with damoctocog alfa pegol and 1.33 (0.00; 2.67) with octocog alfa. Half of the patients receiving damoctocog alfa pegol prophylaxis experienced zero bleeds (n = 9, 50.0%) versus 38.9% (n = 7) of patients treated with octocog alfa. Patients' good quality of life was maintained. CONCLUSION: This study provides routine clinical evidence supporting the benefits of switching from octocog alfa to damoctocog alfa pegol for patients with severe haemophilia A.

Central venous access device insertion and perioperative management of patients with severe haemophilia A: a local experience.

Central venous access device (CVAD) insertion is one of the most common procedures performed on paediatric haemophilia patients. There are no clear guidelines outlining the optimal dosing schedule of factor VIII (FVIII) and duration of treatment required to achieve adequate haemostasis during and after surgery. In this article, we describe the experience at McMaster Children's Hospital using FVIII replacement therapy in 15 children with severe haemophilia A during the course of 7 years. This is a retrospective institutional chart review. Patients between 0 and 18 years of age with severe haemophilia A that underwent CVAD insertion at McMaster Children's Hospital in Hamilton, Ontario, from 2004 to 2010, were identified and charts were reviewed. A total of 15 CVAD insertion surgeries were reviewed. The total average preoperative dose of FVIII was 93.5 IU/kg (range: 53.7-145.4 IU/kg). The total average postoperative dose was 818.7 IU/kg (range: 441-1258 IU/kg). The total perioperative dose was 912.2 IU/kg (range: 495.2-1349 IU/kg). The current study attempts to describe the experience at McMaster Children's Hospital for CVAD insertion surgeries, the average factor dose administered has decreased during the years. These results may be of help in the development of optimal treatment schedules.