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. 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. 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. 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. NCT05036278. The PREDICT study looked at a new way to help treat patients with hemophilia A, a disease that makes it difficult for blood to clot. Usually, patients receive many infusions of standard half-life (SHL) clotting factor products to stop or prevent bleeding; newer extended-half-life (EHL) treatments like damoctocog alfa pegol, allow for fewer infusions. Finding the best treatment plan for each patient is still challenging without thorough pharmacokinetic testing, which shows the body’s effect on the medicine as it gets absorbed, broken down, and removed from the body. To help with this, a scoring system was created that includes things like how many times a patient bleeds, how often they infuse, if they bleed into their joints, their von Willebrand factor level, and how active they are. A total of 21 participants with hemophilia A, who were at least 12 years old and had been getting SHL treatment for at least 6 months, took part in this study. Each participant got a score of low, medium, or high, and followed a special treatment plan with damoctocog alfa pegol for 6 months. The study showed that 70.6% of participants (12 out of 17) had better results, meaning they had fewer bleeds and/or needed fewer infusions compared to their previous treatment. This suggests that using a scoring system to create individual treatment plans can help patients with hemophilia A do better when switching from an SHL to damoctocog alfa pegol.
Quon et al. (Thu,) studied this question.