OC 14.1 - Perioperative management with efanesoctocog alfa in adults, adolescents, and children with severe hemophilia A in the phase 3 XTEND clinical program

Sunday, June 23, 2024

09:30 – 09:45 ICT

Room: Ballroom B4

Presenting Author(s)

Anthony KC Chan, MBBS

Professor
McMaster Children’s Hospital, McMaster University, Hamilton, Canada
Hamilton, Ontario, Canada

Co-Author(s)

SS

Sophie Susen, PhD,MD

Professor of Haematology
Centre Hospitalier Universitaire de Lille, Université de Lille, Lille, France
Lille, Nord-Pas-de-Calais, France
LK

Liane Khoo

Director
Institute of Haematology, Royal Prince Alfred Hospital, Sydney, Australia
Sydney, New South Wales, Australia
Annette von Drygalski, MD, PharmD, RMSK

Professor of Clinical Medicine
University of California San Diego
San Diego, California, United States
Johannes Oldenburg, MD

Professor, Chairman and Director
University Clinic Bonn AöR, Institute of Experimental Haematology and Transfusion Medicine
Bonn, Nordrhein-Westfalen, Germany
MS

Ming-Ching Shen

Haematologist
Division of Hematology-Oncology, Department of Internal Medicine, Changhua Christian Hospital, Changhua 50046, Taiwan
Changhua, Taipei, Taiwan (Republic of China)
Flora Peyvandi, MD, PhD

Full Professor
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Università degli Studi di Milano, Department of Pathophysiology and Transplantation
Milan, Lombardia, Italy
CT

Cristina Tarango

Professor
Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, Ohio, USA
Cincinnati, Ohio, United States
Pratima Chowdary, MD

Professor of Haemophilia, Centre Director
Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital
London, England, United Kingdom
LM

Lara Mamikonian

Clinical Research Director
Sanofi, Cambridge, MA, USA
Cambridge, Massachusetts, United States
HP

Helena Palmborg

Associate Director Scientific Communications
Sobi, Stockholm, Sweden
Stockholm, Stockholms Lan, Sweden
JD

Jennifer Dumont

Senior Medical Director, Global Medical Affairs
Sanofi, Cambridge, MA, USA
Cambridge, Massachusetts, United States
ES

Elena Santagostino

VP, Medicine Development Lead Haemophilia
Sobi, Basel, Switzerland
Basel, Basel-Stadt, Switzerland
Cedric Hermans, MD, FRCP, PhD

Head of the Division of Haematology, the Hemostasis and Thrombosis Unit, and the Hemophilia Center
Saint-Luc University Hospital
Saint-Luc, Brussels Hoofdstedelijk Gewest, Belgium

Background: Efanesoctocog alfa (formerly BIVV001) is a first-in-class high-sustained factor VIII replacement therapy that provides effective bleed prevention with once-weekly dosing (50 IU/kg) in previously treated patients with severe hemophilia A.

Aims: Evaluate efficacy and safety of efanesoctocog alfa for perioperative management in the Phase 3 XTEND clinical program.

Methods: XTEND-1 (NCT04161495) and XTEND-Kids (NCT04759131) studies assessed safety and efficacy of efanesoctocog alfa in previously treated patients ≥12 and < 12 years old, respectively, with severe hemophilia A. Participants from each study could continue treatment in the XTEND-ed study (NCT04644575). Participants provided informed consent, and studies were approved by ethics committees. Participants undergoing surgery were to receive a pre-operative loading dose of efanesoctocog alfa 50 IU/kg. For major surgeries, post-operative doses of 30 or 50 IU/kg every 2–3 days were allowed. Surgery endpoints included dose, number of injections, hemostatic response, factor consumption, blood loss, and blood transfusions during the perioperative period. Interim data cut: January 17, 2023.

Results: Forty-one participants ( < 18 years, n=9; ≥18 years, n=32) underwent 49 major surgeries (Table 1); 23 were orthopedic surgeries, with knee arthroplasty most common (n=12). All single 50 IU/kg pre-operative doses maintained hemostasis during major surgery. Median (range) number of doses per major surgery was 4.0 (1–7) during the perioperative period (Days −1 to 14). Hemostatic response was rated excellent for 43 surgeries and good for 5 surgeries. Median (range) blood loss was 20 (0–1000) mL during surgery (n=29); red blood cell transfusion was required for 1 total knee replacement. Of 32 minor surgeries (Table 2) among 28 participants, 27 were managed with 1 pre-operative dose and 5 with no pre-operative dose. Of those with an assessment (n=25), all hemostatic responses were rated excellent.

Conclusion(s): Efanesoctocog alfa was effective and well tolerated for perioperative management in participants with severe hemophilia A.
Funded by Sanofi and Sobi.