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

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.