A New Risk-Based Scoring Approach to Individualize Prophylaxis in Patients with Hemophilia A: Results from the PREDICT Study.

INTRODUCTION

There is currently no consensus on optimal prophylaxis with clotting factor concentrates in hemophilia A. With or without pharmacokinetic data, prophylaxis regimens are often based on local practice and patient-specific factors, including bleeding history and activity level. The PREDICT study evaluated a baseline clinical risk score, based on phenotypic and biologic variables, to guide prophylactic regimen selection when switching from standard half-life (SHL) factor VIII concentrates to damoctocog alfa pegol.

METHODS

This multicenter, prospective study enrolled 21 participants aged ≥ 12 years with congenital hemophilia A of any severity who had received SHL prophylaxis for ≥ 6 months. Each participant was assigned a risk score (low, medium, high) based on five predefined variables: bleeding phenotype, treatment frequency, active target joints, von Willebrand factor levels, and physical activity. All participants initiated damoctocog alfa pegol at twice-weekly dosing for 4 weeks, after which regimens were individualized: 2×/week for high risk, every 5 days (Q5D) for medium risk, and Q5D for 4 weeks followed by less frequent dosing for low risk participants. A favorable outcome was defined as a reduction in annualized bleeding rate (ABR) and/or infusion frequency compared with previous SHL prophylaxis.

RESULTS

Among 17 evaluable participants, 12 (70.6%) achieved a favorable outcome for treated and untreated bleeds. All 12 had improved ABR, and 11 required fewer infusions. When considering only treated bleeds, 77% achieved a favorable outcome. Mean ABR decreased by 8.01 and infusion frequency reduced by a mean of 6.9 infusions/month. The 25 active target joints identified at baseline were reduced by 96% at study completion.

CONCLUSIONS

The PREDICT risk-based scoring system-guided individualized prophylaxis with damoctocog alfa pegol, yielding improved bleeding outcomes and reduced treatment burden in most participants. These findings support the use of structured, risk-adapted approaches to personalize prophylaxis when transitioning from SHL to damoctocog alfa pegol.

TRIAL REGISTRATION: NCT05036278.