Pain Reduction Following Eptacog Beta Treatment of Bleeding Episodes in Adolescents and Adults With Haemophilia A or B Complicated by Inhibitors

Authors: Buckner, TW; Kessler, C; Castaman, G; Hermans, C; Jiménez-Yuste, V; Mahlangu, J; Miesbach, W; Oldenburg, J; Recht, M; Valentino, LA; Wheeler, AP; Pipe, SW

Affiliations: Hemophilia and Thrombosis Center, University of Colorado School of Medicine, Aurora, Colorado, USA. Lombardi Comprehensive Cancer Center, MedStar Georgetown University Hospital, Washington, District of Columbia, USA. Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy. Division of Hematology, Cliniques Saint-Luc, Université Catholique de Louvain (UCLouvain), Brussels, Belgium. Hospital Universitario La Paz Idi-Paz, Autónoma University, Madrid, Spain. Department of Molecular Medicine and Haematology, Faculty of Health Sciences, University of the Witwatersrand and NHLS, Johannesburg, South Africa. Goethe University Hospital, Frankfurt, Germany. Institute of Experimental Haematology and Transfusion Medicine, Medical Faculty, University Clinic Bonn AöR, University of Bonn, Bonn, Germany. Yale Center for Bleeding and Clotting Disorders, Yale University School of Medicine, National Bleeding Disorders Foundation, New York, New York, USA. Rush University Medical Center, Chicago, Illinois, USA. Washington Center for Bleeding Disorders, Division of Pediatric Hematology/Oncology & Division of Hematology and Oncology, University of Washington School of Medicine, Seattle, Washington, USA. University of Michigan, Ann Arbor, Michigan, USA.

Publication: Haemophilia;2025. e70077

ABSTRACT: INTRODUCTION: Joint and muscle bleeding in persons with haemophilia (PwH) causes acute pain, an important patient-reported outcome for evaluating treatment response. Eptacog beta is a recombinant activated human FVII bypassing agent approved for the treatment and control of bleeding in PwH A or B with inhibitors (PwHABI) ≥12 years using initial dose regimens (IDRs) of 75 or 225 μg/kg. AIM: To evaluate pain reduction in PwHABI who treated bleeding episodes (BEs) with eptacog beta during the PERSEPT 1 trial. METHODS: PwHABI were randomly assigned to 75 or 225 μg/kg IDRs for BE treatment. Participants reported pain levels at predefined treatment evaluation timepoints using a visual analogue scale (VAS). Pain analyses were stratified by age, IDR assignment, and baseline pain levels. The diagnostic potential for using VAS pain score reductions to assess treatment response was explored using receiver operating characteristic (ROC) analysis. RESULTS: Five adolescents and 22 adults treated 468 BEs. Mean VAS scores decreased 30%-58% from baseline at 3 h post-initial eptacog beta infusion amongst age and IDR categories, with further VAS score reductions observed through 24 h. PwHABI with high baseline BE pain showed larger absolute VAS score reductions before ending treatment than PwHABI experiencing mild BE pain. The optimum VAS score percentage reduction cutoff for predicting treatment outcomes was 44%. CONCLUSION: Pain relief over 24 h was observed with eptacog beta treatment across IDR and age categories. This post hoc study demonstrates that percentage reduction from baseline VAS score influences perception of bleed resolution and can predict treatment outcomes.