Observational Study of Pegylated rVIII Administration in Real Clinical Practice
A recently published article by American hematologists provided valuable information about the treatment with pegylated recombinant factor VIII (rVIII) in real clinical practice. This is the most extensive observational study to date that evaluates the clinical effect of the treatment and at the same time compares the consumption of FVIII in people with hemophilia A who have changed medication from normal half-life (SHL) or extended half-life (EHL) concentrates to pegylated rVIII.
Introduction
Rurioctocog alfa pegol (Adynovate in North America, Adynovi in Europe) represents a new, third-generation rFVIII with extended half-life (EHL). It is a recombinant FVIII molecule that is modified by pegylation. This structural modification of the drug results in a 1.5 times longer biological half-life compared to SHL products.
Results of the American Study
Initial Information on Patients Before Treatment Change
Data were collected from a group of 56 individuals with hemophilia A, with an average age of 26 years (range 5–88 years). 20% of the hemophiliacs were under 12 years old, and 89% of them had a severe form of the disease. All individuals were treated with FVIII concentrates (SHL or EHL) for at least one year before switching to Adynovate. Prior to the change, 73% received prophylaxis, while the rest were treated on-demand during bleeding episodes. During the preceding period, the observed individuals experienced recurrent bleeding into an average of 1.8 joints (target joints). 68% of the patients reported mild to moderate pain at the initial examination.
Benefits of the New Treatment
Before the change, the average frequency of dose application was 2.8 times per week for patients treated with SHL and 1.8 for those treated with EHL products. After switching to Adynovate, patients used 2.2 doses per week. The median duration of prophylaxis with Adynovate was 12 months, following a previous period of 80.8 months for patients treated with SHL and 13.5 months for those treated with EHL concentrates.
The average annual bleeding rate (ABR) was 5.9 with the previous SHL prophylaxis and 4.7 with the use of EHL products. Thanks to Adynovate, the average ABR decreased by 71% (from 5.8 to 1.7; p < 0.001). For 20 hemophiliacs, no bleeding was observed after switching to Adynovate. The number of days per week requiring factor administration decreased by 20.9% (p < 0.001). In 47 hemophiliacs previously using SHL products, the weekly factor dose slightly reduced from 109.8 to 100.6 IU/kg (p = 0.094).
The proportion of patients with good or complete adherence to treatment increased from 68% to 80% after switching to pegylated rVIII.
Conclusion
The switch from SHL and EHL rFVIII concentrates to pegylated rVIII in the American patient population was associated with fewer bleeding episodes. Data from real clinical practice indicate more effective prophylaxis and better adherence to treatment. In patients who switched to pegylated rVIII from previous SHL concentrate treatments, a reduction in factor consumption was documented. Pegylated rVIII products thus become an attractive option for prophylactic treatment in hemophilia A.
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Source: Aledort L., Milligan S., Watt M., Booth J. A retrospective observational study of rurioctocog alfa pegol in clinical practice in the United States. J Manag Care Spec Pharm 2020; 26 (4): 492–503, doi: 10.18553/jmcp.2020.26.4.492.
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