Individualized Prophylaxis with BAY 81-8973: Clinical Experience in an Italian Patient with Severe Hemophilia A

Abstract

Introduction: Prophylaxis with a factor VIII (FVIII) product is the standard of care for patients with severe hemophilia A to prevent bleeding episodes and development of target joints and hemophilic arthropathy. In patients with established joint disease, prophylaxis can slow progression of joint damage, which can improve health-related quality of life. Adherence to prophylaxis is critical to its success. Missing or skipping a prophylaxis infusion can cause FVIII levels to fall below the desired target level, which can increase the risk of breakthrough bleeding episodes. To promote adherence to prophylaxis, healthcare providers and patients must work together to ensure that the prophylaxis regimen is manageable for each patient. In this case report, we describe the strategy we used to improve adherence, including changes to the prophylaxis dosing regimen, in a patient with severe hemophilia A.Demographics and medical history: A 54-year-old patient was diagnosed with severe hemophilia A in early childhood and treated on demand with a plasma-derived FVIII product, despite meeting criteria for use of prophylaxis. In 2000, he began tertiary prophylaxis with a standard-acting recombinant FVIII (rFVIII) product given twice weekly. At that time, he had target joints in both knees and both elbows and a Hemophilia Joint Health Score (HJHS) of 16. In 2007, he underwent surgical replacement of the left knee. In 2010, prophylaxis with a rFVIII was started at 3000 IU (32 IU/kg) 3 times per week. The patient's job as a vendor in outdoor markets affected his ability to adhere to this prophylaxis regimen. To facilitate adherence, prophylaxis dosing was decreased to twice weekly. The patient's comorbidities included obesity and hepatitis C infection, which did not respond to combined treatment with interferon and ribavirin.Current prophylaxis treatment: In 2015, because of weight gain (to 110 kg), joint bleeding episodes (2-3 per year), repeated occurrence of left ankle pain, and further joint deterioration (HJHS score, 25), prophylaxis dosing was increased to 4000 IU (40 IU/kg) 3 times per week. Adherence to this regimen was again difficult for the patient. In March 2017, based on pharmacokinetic results obtained in this patient with BAY 81-8973 (Kovaltry®, Bayer, Berkeley, CA; Table 1), he was transitioned to BAY 81-8973 5000 IU (45 IU/kg) twice weekly (every 84 hours, on Monday morning and Wednesday evening). This prophylaxis regimen accommodated his work schedule, and no bleeding episodes occurred after the patient started BAY 81-8973 prophylaxis.Conclusion: This case illustrates that the prophylaxis dose and dosing interval can be individually tailored, keeping in mind the clinical goal of treatment and the patient's bleeding phenotype and daily activities. In this patient, tailored prophylaxis dosing with BAY 81-8973 was key to improved adherence, outcomes, and health-related quality of life. [Display omitted] DisclosuresNo relevant conflicts of interest to declare.