Turoctocog alfa pegol provides effective management for major and minor surgical procedures in patients across all age groups with severe haemophilia A: Full data set from the pathfinder 3 and 5 phase III trials

Authors: Tosetto, A., Neff, A., Lentz, S. R., Santagostino, E., Nemes, L., Sathar, J., Meijer, K., Chowdary, P., Shen, C., Landorph, A., and Hampton, K.

Publication: Haemophilia; April 2020

Affiliations: Hematology Department, Haemophilia and Thrombosis Center, San Bortolo Hospital, Vicenza, Italy; Department of Hematology/Oncology, Cleveland Clinic, Cleveland, OH, USA; Division of Hematology, Oncology, and Blood & Marrow Transplantation, Department of Internal Medicine, University of Iowa Carver College of Medicine, Iowa City, IA, USA; Fondazione IRCCS Ca Granda – Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Haemophilia and Thrombosis Center, Milan, Italy; National Haemophilia Centre and Haemostasis Department, Medical Centre of Hungarian Defence Forces, Budapest, Hungary Department of Haematology, Ampang Hospital, Selangor, Malaysia; Department of Haematology, University Medical Centre Groningen, University of Groningen, Groningen, The Netherlands; Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, UK; Novo Nordisk A/S, Bagsvaerd, Denmark

Abstract: INTRODUCTION: Turoctocog alfa pegol is a glycoPEGylated recombinant factor VIII (FVIII) with an extended half-life developed for prophylaxis, treatment of bleeds and perioperative management in patients with haemophilia A. AIM: Evaluate the efficacy and safety of turoctocog alfa pegol treatment for major and minor surgeries in the pathfinder 3 and 5 phase III trials. METHODS: Adults/adolescents aged >/=12 years with severe haemophilia A (FVIII <1%) received perioperative turoctocog alfa pegol treatment planned to achieve FVIII activity levels >80% during major surgery (pathfinder 3). The primary end point was haemostatic efficacy during surgery; secondary end points were blood loss, haemostatic effect postsurgery, consumption, transfusions, safety and health economics. Children (0-11 years) undergoing minor surgeries received 20-75 IU/kg turoctocog alfa pegol at Investigator’s discretion (pathfinder 5). RESULTS: pathfinder 3 included 35 patients undergoing 49 major surgeries. Haemostasis was successful in 47/49 (95.9%) surgeries; two had moderate haemostatic responses. Median (mean) blood loss during major surgery was 75 (322.6) mL. Four bleeds were reported postsurgery; three were successfully treated with turoctocog alfa pegol (one was not evaluated). On the day of surgery, overall mean (median) dose was 75.5 (74.5) IU/kg and mean (median) number of doses was 1.7 (2.0). Five procedures required 11 transfusions on the day of surgery or days 1-6. No safety concerns or inhibitors were identified. Forty-five minor surgeries in 23 children were performed without complications. CONCLUSION: Turoctocog alfa pegol was effective for perioperative haemostatic management of major and minor surgeries in patients across age groups with severe haemophilia A.