This review published in EFORT Open Reviews detailed how management of hemophilia among children requires a team approach, of which the patient and the family are a part because compliance has ultimate importance.
Hemophilia often targets large joints of the lower extremity and elbow, and repeated hemophilic episodes can lead to “subsequent degenerative arthritis with pain, limited range of motion (ROM), deformity and severe contractures.” Patients with severe hemophilia may experience spontaneous bleeds once or twice per week juxtaposed with patients with a moderate or mild form of the disease. Hemophilic children with joints which bleed frequently suffer a gradual loss of sharing activities with their friends and cannot maintain their quality of life and eventually become disabled in the disease’s end stages, which are characterized by fibrous contracture, loss of joint space and substantial disorganization of the joint structures, and pain due to degenerative arthritis.
Concentrated fVIII enables primary prevention and replacement therapy, and while preventative treatment serves as a key component to prevent disease progression, such treatments are expensive, and pediatric patients often develop resistance to regular injections and require venous access. This is the key to prevent clinical deterioration of the disease; however, it is expensive and frequently cannot be followed in many countries due to economic reasons. Additionally, many patients develop decreased compliance with regular injections for infusion of factor and its inherent need for venous access. The review notes, “about 15% to 20% of patients with hemophilia develop an inhibitor antibody that prevents the clotting factor from being able to develop blood clots and stop bleeding. Different kinds of clotting factors are then needed to by-pass the normal clotting sequence and induce clotting.”
Fractures are less frequent in hemophilia disease, due to patients’ protected lifestyles and lower level of activities. However, for such patients arthropathy should still be considered, especially for fractures around joints that have significant loss of motion. Many hemophilic patients experience multiple hemarthrosis episodes in a target joint and develop severe joint degeneration as early as their teens. End-stage hemophilic arthropathy in the hip or knee joint can be successfully treated with total joint arthroplasty. However, the success rate compared with a primary arthritis cohort may not be as good, and certain pre-operative knee characteristics may affect the functional results. Also, existing joint deterioration often requires bone grafting and specific components such as stems, augments or wedges. Associated hip and ankle arthritis with deformities are frequently addressed with additional surgery.
“Hemophilia management requires a team approach, of which the patient and the family are a part because compliance has ultimate importance,” the authors wrote in their conclusion. “With the administration of National Joint Registries, every child with hemophilia must be followed closely with regular multi-team visits.”
They added that “effective collaboration with orthopedic departments and hemophilia services and societies have a significant contributing role in this lifelong management.”