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.
The participant or legally authorized representative is willing and able to provide written informed consent.
Diagnosis of severe hemophilia A (factor VIII activity < 1%).
Male sex.
Age between 30 and 50 years (inclusive).
Unwillingness of the participant, parent, or legally authorized representative to provide informed consent.
Diagnosis of a bleeding disorder other than or in addition to severe hemophilia A.
Active Factor VIII inhibitors at the time of enrollment
History of a disease known to influence bone metabolism unrelated to a bleeding disorder. (Examples: Paget's disease, osteogenesis imperfecta, Ehlers Danlos syndrome, Hyperparathyroidism)
Patients that are seen at dedicated Hemophilia treatment Centers
Case-only
Prospective
Not listed
Longitudinal change in femoral neck bone mineral density (g/cm²)
Bone mineral densitometry
Longitudinal change in Lumbar spine (L1-L4) bone mineral density (g/cm²)
Bone mineral densitometry
Longitudinal change in total hip bone mineral density (g/cm²)
Bone mineral densitometry
Longitudinal Change in Bone Remodeling Biomarkers and Cytokines
PINP, CTX-I, OPG, RANKL, IL-1β, IL-6, and TNF-α
Change in Thrombin Generation and Plasmin Generation Parameters
Simultaneous Thrombin and Plasmin Generation Assay
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Arkansas Children's Hospital Research Institute
Lead sponsor
Genzyme, a Sanofi Company
Collaborator
