State of care for hemophilia in pediatric patients

On demand treatment of bleeding episodes is still the main approach to hemophilia care of patients of any age. Prompt infusion of coagulation factor concentrate in the home setting allows treatment of hemorrhages at early onset, reducing the incidence of complications and improving the quality of life. Nevertheless, the technological evolution and progressive improvement in the safety of therapeutic products have changed the management of the disease, particularly in children. The current availability of safer concentrates has drastically reduced the risk of transmission of blood-borne infections. Innovative approaches, such as early primary prophylaxis and immune tolerance induction, have become feasible and their introduction represents a major advance in the achievement of the main therapeutic goals: control of the bleeding diathesis and elimination of inhibitors. Prophylactic regimens have been shown to be effective in preventing the occurrence not only of joint bleeding but also of arthropathy when started early in children with severe hemophilia. Inhibitor development still represents the main complication of hemophilia treatment, making concentrate administration ineffective. Immune tolerance induction by daily infusion of coagulation factor concentrate was shown to eradicate the inhibitors in 63 to 83% of patients. These intensive treatment regimens are administered at home to very young children and create the problem of adequate venous access. Subcutaneous venous ports have been used in patients with hemophilia if peripheral veins could not be frequently accessed; however, the risk of infection is an important limit to their use.