State of Care for Hemophilia in Pediatric Patients

Abstract

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.

Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group

“…5 The overall prevalence of inhibitors in patients with hemophilia A or B is estimated to be approximately 9% and 3%, respectively; patients with moderately severe (factor levels 1%-5%) and severe hemophilia A (less than 1%) have a greater risk for inhibitors; reported prevalences for them are between 7% and 20%. 6 Special therapeutic approaches are used in patients with inhibitors to reduce antibody levels, 7,8 such as the induction of immune tolerance 9,10 and plasma exchange with or without immunoadsorption on protein A columns. For the actual treatment of bleeding episodes, the main therapeutic approaches are large doses of human factor VIII (FVIII), FVIII derived from porcine plasma and products that bypass the coagulation defect, such as prothrombin complex concentrates (PCCs), activated prothrombin complex concentrates (aPCCs), [11][12][13][14] and recombinant activated factor VII (rFVIIa).…”

Patients’, physicians’, and pharmacists’ preferences towards coagulation factor concentrates to treat haemophilia with inhibitors: results from the COHIBA Study

“…In addition, not all patients are considered suitable candidates for ITI; for example, patients with long-standing inhibitors, those with further difficulties with treatment, those at an increased risk of serious bleeding complications and those with chronic joint diseases. Bypassing agents such as prothrombin complex concentrates (PCC), activated prothrombin complex concentrates (APCC) and recombinant activated factor VII (rFVIIa) have been successfully adopted in a number of these challenging situations, including emergency, home treatment and surgery [4,[25][26][27][28][29][30][31][32][33][34][35][36][37], in which they contribute to prolonging patientsÕ life expectancy and improving quality of life [38]. However, no consensus exists yet on the therapeutic strategy and products to use in order to improve the management of inhibitor patients.…”

Psycho‐social determinants of quality of life in children and adolescents with haemophilia—a cross‐cultural approach

“…The availability of factor concentrates has largely expanded the length and the quality of the lives of these patients. Early diagnosis is required to provide factor replacement therapy timely in children (Santagostino, Gringeri, & Mannucci, 2002) from the fi rst bleeding event onwards, which most of the time-depending on the severity of the disease-occur within the fi rst life year (Liesner, Khair, & Hann, 1996). Dependent on the economic situation of a specifi c country and their treatment guidelines, administration includes different treatment modalities and dosage of factor concentrate.…”