Arkansas Children's Hospital Research Institute
This study will compare bone mineral density in patients with severe hemophilia A receiving prophylaxis with emicizumab or efanesoctocog alfa. Participants will undergo assessments of bone mineral density, bone remodeling biomarkers, thrombin generation, plasmin generation, and joint health over a five-year period. The study aims to evaluate whether differences in prophylactic therapy are associated with differences in bone health outcomes.
Reduced bone mineral density, osteoporosis, and fractures are increasingly recognized in persons with severe hemophilia A. The mechanisms underlying impaired bone health in hemophilia are multifactorial and may include reduced physical activity, chronic joint disease, inflammation, and abnormalities in coagulation-related pathways involved in bone remodeling. Thrombin has been shown to play an important role in bone metabolism through activation of protease-activated receptor-1 (PAR-1) signaling pathways that influence osteoblast and osteoclast activity. Reduced thrombin generation in severe…
Inclusion Criteria: 1. The participant or legally authorized representative is willing and able to provide written informed consent. 2. Diagnosis of severe hemophilia A (factor VIII activity \< 1%). 3. Male sex. 4. Age between 30 and 50 years (inclusive). 5. BMI between 18.5 and 40 kg/m2 6. The participant must have been on prophylaxis with Efanesoctocog alfa or Emicizumab for at least 3 months prior to enrollment and intend to remain on the current regimen for the next 5 years. 7. Willingness to undergo all research procedures, including DEXA scans and the collection of blood samples. 8. Wil…
Arctuva estimate
Verify or correct the compensation shown for this listing.