Current Issues in Prophylactic Therapy for Persons with Hemophilia

Abstract

Factor VIII or IX replacement in a prophylactic manner is utilized for many patients with moderate to severe hemophilia A or B. Studies have shown it to be effective in reducing or preventing degenerative joint disease in many but not all patients. However, many unanswered questions still exist and optimization of this expensive treatment regimen is needed. This paper recounts the current products that are available for use and explores the literature regarding different treatment regimens. It explores age at initiation, dose, interval between infusions, joint health outcomes, barriers to compliance and age at discontinuation of prophylaxis. Individualized treatment is recommended. Collaborative efforts are needed to improve outcomes for all persons with hemophilia.

A 5-Month-Old Male With an Isolated Prolonged Partial Thromboplastin Time

“…Recombinant Factor VIII products currently available include Recombinate (first generation), Helixate/Kogenate FS and ReFacto (second generation), and Advate (third generation). 4 There is only one recombinant Factor IX product commercially available and this is marketed as BeneFIX. 4 For patients with mild to moderate hemorrhages such as those involving the oral and nasal cavities, the goal is to raise Factor VIII levels to at least 30% to 40% and Factor IX levels to at least 30%.…”

“…4 There is only one recombinant Factor IX product commercially available and this is marketed as BeneFIX. 4 For patients with mild to moderate hemorrhages such as those involving the oral and nasal cavities, the goal is to raise Factor VIII levels to at least 30% to 40% and Factor IX levels to at least 30%. 5 When bleeding episodes involve the cranium, bowels, or airways, aggressive factor replacement is imperative.…”

“…This concept was developed in Sweden in 1958. 4 Since then, the prophylactic use of Factor VIII and IX concentrates has become an integral part of the management of hemophiliacs, particularly in developed countries. Studies have shown that many patients benefit from prophylaxis (versus replacing deficient factors only when patients are bleeding) in that it allows patients to have a more normal lifestyle by preventing the majority of bleeding episodes and preventing joint disease secondary to hemarthroses.…”

“…One school of thought advocates that prophylaxis be instituted during the second year of life when the patient is not bleeding or after the first joint bleed, whichever comes first. 4 It has been shown that early prophylaxis may reduce the frequency of bleeding into joints. 6 However, early prophylaxis may increase the overall cost of therapy and may promote the development of inhibitors.…”

“…6 However, early prophylaxis may increase the overall cost of therapy and may promote the development of inhibitors. 4 Moreover, since 10% to 14% of patients with severe hemophilia rarely have bleeding episodes, these patients may be over-treated by this early approach. 4 The second school of thought believes that hemophiliacs should be started on prophylaxis only after they have developed a pattern of bleeding.…”