Steven R Lentz1, Dragana Janic2, Kaan Kavakli3, Predrag Miljic4, Johannes Oldenburg5, Margareth C Ozelo6, Elena Santagostino7, Takashi Suzuki8, Silva Zupancic Šalek9,10,11, Lars Korsholm12, Irina Matytsina12, Andreas Tiede13. 1. Division of Hematology, Oncology and Blood & Marrow Transplantation, Internal Medicine, The University of Iowa Carver College of Medicine, Iowa City, Iowa. 2. School of Medicine, University Children's Hospital, University of Belgrade, Belgrade, Serbia. 3. Department of Pediatric Hematology, Children's Hospital, Ege University Faculty of Medicine, Izmir, Turkey. 4. School of Medicine, Clinic of Hematology, Clinical Center of Serbia, University of Belgrade, Belgrade, Serbia. 5. Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany. 6. IHTC 'Claudio L.P. Correa', INCT do Sangue Hemocentro UNICAMP, University of Campinas, São Paulo, Brazil. 7. Foundation IRCCS Ca' Granda, Maggiore Hospital Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy. 8. Tokyo Medical University, Tokyo, Japan. 9. University Hospital Centre Zagreb, Zagreb, Croatia. 10. Medical School of Zagreb, Zagreb, Croatia. 11. Medical School of Osijek, Osijek, Croatia. 12. Novo Nordisk A/S, Søborg, Denmark. 13. Hannover Medical School, Hannover, Germany.
Abstract
INTRODUCTION: Turoctocog alfa is a recombinant factor VIII (FVIII) molecule, approved for treatment and prophylaxis of bleeding in patients with haemophilia A. In the guardian 1 (adolescents/adults) and guardian 3 (children) phase 3 trials, turoctocog alfa demonstrated a favourable efficacy and safety profile. Guardian 1 or 3 completers could enrol in the guardian 2 extension. Final guardian 2 results are reported here. AIM: Investigate long-term safety and efficacy of turoctocog alfa administered for prophylaxis and treatment of bleeds. METHODS: In this phase 3b open-label trial, previously treated males of all ages with severe haemophilia A received prophylaxis regimens of turoctocog alfa or on-demand treatment of bleeds. The primary safety endpoint was frequency of FVIII inhibitor development. Efficacy endpoints included annualized bleeding rate (ABR) during prophylaxis, haemostatic response in treatment of bleeds and number of injections required to treat bleeds. RESULTS: Overall, 213 patients were dosed with turoctocog alfa; 207 patients received prophylaxis; 19 received on-demand treatment. No FVIII inhibitors (≥0.6 BU) were reported. For all patients on prophylaxis, overall median ABR was 1.37 bleeds/y; success rate for treatment of bleeds was 90.2%; and 88.2% of bleeds were controlled with 1-2 injections of turoctocog alfa. For the on-demand regimen, overall median ABR was 30.44 bleeds/y; success rate for treatment of bleeds was 96.7%; and 94.9% of bleeds were controlled with 1-2 injections of turoctocog alfa. CONCLUSION: Extended use of turoctocog alfa is safe and effective for prevention and treatment of bleeding episodes in previously treated patients with haemophilia A across all ages.
INTRODUCTION:Turoctocog alfa is a recombinant factor VIII (FVIII) molecule, approved for treatment and prophylaxis of bleeding in patients with haemophilia A. In the guardian 1 (adolescents/adults) and guardian 3 (children) phase 3 trials, turoctocog alfa demonstrated a favourable efficacy and safety profile. Guardian 1 or 3 completers could enrol in the guardian 2 extension. Final guardian 2 results are reported here. AIM: Investigate long-term safety and efficacy of turoctocog alfa administered for prophylaxis and treatment of bleeds. METHODS: In this phase 3b open-label trial, previously treated males of all ages with severe haemophilia A received prophylaxis regimens of turoctocog alfa or on-demand treatment of bleeds. The primary safety endpoint was frequency of FVIII inhibitor development. Efficacy endpoints included annualized bleeding rate (ABR) during prophylaxis, haemostatic response in treatment of bleeds and number of injections required to treat bleeds. RESULTS: Overall, 213 patients were dosed with turoctocog alfa; 207 patients received prophylaxis; 19 received on-demand treatment. No FVIII inhibitors (≥0.6 BU) were reported. For all patients on prophylaxis, overall median ABR was 1.37 bleeds/y; success rate for treatment of bleeds was 90.2%; and 88.2% of bleeds were controlled with 1-2 injections of turoctocog alfa. For the on-demand regimen, overall median ABR was 30.44 bleeds/y; success rate for treatment of bleeds was 96.7%; and 94.9% of bleeds were controlled with 1-2 injections of turoctocog alfa. CONCLUSION: Extended use of turoctocog alfa is safe and effective for prevention and treatment of bleeding episodes in previously treated patients with haemophilia A across all ages.
Authors: Andreas Tiede; Faraizah Abdul Karim; Victor Jiménez-Yuste; Robert Klamroth; Sandra Lejniece; Takashi Suzuki; Andreas Groth; Elena Santagostino Journal: Haematologica Date: 2021-07-01 Impact factor: 9.941
Authors: Robert Klamroth; Clemens Feistritzer; Ute Friedrich; Steven R Lentz; Kirsten Reichwald; Marek Zak; Pratima Chowdary Journal: J Thromb Haemost Date: 2019-11-15 Impact factor: 5.824
Authors: Carmen Escuriola Ettingshausen; Olga Katsarou; Barbara Faganel Kotnik; Annie Borel Derlon; Rudolf Schwarz; Paula F Ypma; Irina Matytsina; Sohan Dey; Roger E G Schutgens Journal: Haemophilia Date: 2021-11-17 Impact factor: 4.263