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  • Writer's pictureThe Rare360 Editorial Team

Assessing Safety and Effectiveness of Damoctocog Alfa Pegol in Hemophilia A Patients

Laboratory result with blood tubes

Key Highlights from the HEM-POWR Study

  • A multinational, 33-month observational study, HEM-POWR, assesses damoctocog alfa pegol's effectiveness and safety in patients with hemophilia A.

  • Damoctocog alfa pegol is an extended half-life (EHL) recombinant factor VIII product for prophylactic, on-demand, and perioperative treatment.

  • The study covers a diverse group of patients with mild, moderate, and severe hemophilia A, providing a real-world perspective.

  • Key findings include reduced annualized bleeding rates, improved joint health, and no inhibitor development.

  • The results support damoctocog alfa pegol as an effective and well-tolerated treatment for hemophilia A, offering dosing flexibility in a real-world clinical setting.

Hemophilia A: A Bleeding Disorder

Hemophilia A, a rare congenital bleeding disorder, is characterized by a deficiency in coagulation factor VIII (FVIII). The severity of hemophilia A varies, classified based on FVIII activity levels into severe (FVIII <1%), moderate (FVIII 1%–5%), and mild (FVIII >5%–40%). Patients with hemophilia A often experience spontaneous bleeding into joints and muscles, which can lead to joint damage and life-threatening bleeding. The mainstay of treatment has been prophylactic replacement therapy with clotting FVIII concentrate, shown to improve joint health and quality of life.

Damoctocog Alfa Pegol: A Promising Treatment

Damoctocog alfa pegol (sold as Jivi®) is an extended half-life (EHL) recombinant FVIII product. It has been designed to prolong its half-life by conjugating it with polyethylene glycol (PEGylated). This unique formulation provides the potential for longer intervals between dosing, reducing the frequency of FVIII infusions without compromising therapeutic effectiveness, compared to standard half-life (SHL) products.

The HEM-POWR Study: Promising Insights for Hemophilia A Patients

The HEM-POWR study, an international undertaking, explores the efficacy and safety of damoctocog alfa pegol in individuals with hemophilia A who have received previous treatment (PTPs). This groundbreaking research, published in the European Journal of Hematology in October 2023, encompasses 63 participating centers across 14 countries in Europe, the Americas, and Asia as of August 2022.

Key Findings of the Study

The latest interim analysis, conducted at 33 months, provides valuable insights into the use of damoctocog alfa pegol. It demonstrates that this EHL product is a well-tolerated and effective treatment option in a diverse group of hemophilia A patients, including those with mild, moderate, and severe disease. The study found several noteworthy outcomes:

  • Reduced Bleeding Incidents: Patients receiving damoctocog alfa pegol experienced a decrease in annualized bleeding rates (ABRs). The ABR for all bleed types decreased significantly during the observation period.

  • Improved Joint Health: The study showed a decrease in the number of patients with affected joints, suggesting an improvement in joint health over time. This is a critical outcome, as joint damage is a significant concern for individuals with hemophilia.

  • Safety and Tolerability: Damoctocog alfa pegol was well-tolerated, with no study drug-related adverse events reported. Importantly, no inhibitor development occurred during the study.

  • Resource Utilization: Data on FVIII product usage indicated that patients on damoctocog alfa pegol had a reduced number of infusions compared to their prior FVIII product. This suggests that damoctocog alfa pegol may provide effective prophylaxis with fewer infusions, potentially improving patients' treatment experience.

Comprehensive view of the proportion of patients with affected joints during the HEM-POWR study, categorized by the analysis time points and the severity of hemophilia A.
Comprehensive view of the proportion of patients with affected joints during the HEM-POWR study, categorized by the analysis time points and the severity of hemophilia A.

Contribution to Hemophilia Treatment

These findings contribute to the growing body of evidence supporting the effectiveness and safety of damoctocog alfa pegol in real-world clinical practice. The study emphasizes the significance of personalized and flexible treatment options, offering dosing regimens tailored to individual patient needs, while still ensuring protection against bleeding events and joint damage.

In conclusion, the HEM-POWR study, published in the European Journal of Hematology in October 2023, provides substantial real-world evidence that damoctocog alfa pegol is a valuable treatment option for patients with hemophilia A, addressing the challenges associated with this rare bleeding disorder. It offers hope for improved management, fewer bleeds, better joint health, and enhanced overall quality of life for individuals living with hemophilia A.


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