Model of Risk of Bleeding Associated with Physical Activity in Hemophiliacs with Factor and Non-Factor Treatment
In the treatment of hemophilia A, both FVIII concentrate replacement therapy and non-factor therapy are available today. However, there is no clinical data comparing the risk of bleeding associated with physical activity between factor and non-factor treatments. In this situation, one approach is to model possible scenarios and attempt to indirectly compare different modalities of bleeding prevention.
In a study published in the journal Expert Review of Hematology, microsimulation using computer modeling was employed to estimate the risk of bleeding associated with physical activity under various types of treatment. Rurioctocog alfa pegol was utilized as a representative of factor treatment, and emicizumab for non-factor treatment.
For the pharmacokinetic (PK) model of rurioctocog alfa pegol, data from individual patients from the PROPEL study were used, where prophylaxis targeted two different minimum levels: the group with a target minimum level of 1−3% and another group with a target level of 8−12%. For emicizumab, the authors assumed 20% FVIII activity equivalence (estimated based on in vitro and in vivo data).
Six hypothetical patient profiles with varying levels of physical activity (according to the National Hemophilia Foundation risk scale) were created. For each profile and different treatment regimen, the total risk of bleeding over 2 weeks was estimated, which was referenced to a patient engaging in low-intensity activity with a 0% FVIII level.
This microsimulation demonstrated that, for the evaluated activity profiles, prophylaxis with rurioctocog alfa pegol in both target groups was associated with a lower risk of bleeding compared to emicizumab (p < 0.001).
In conclusion, the authors state that while this model does not work with the results of real patients, it suggests that PK-guided rurioctocog alfa pegol prophylaxis may reduce the risk of bleeding during physical activity.
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Source: Sun S. X., Crawford S. Microsimulation to compare activity-related bleed risks between pharmacokinetic-guided rurioctocog alfa pegol prophylaxis and emicizumab. Expert Rev Hematol 2023 Mar; 16 (3): 205−211, doi: 10.1080/17474086.2023.2162498.
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