Novel therapies and current clinical progress in hemophilia A

Abstract

The evolution of hemophilia treatment and care is a fascinating one but has been fraught with many challenges at every turn. Over the last 50 years or so patients with hemophilia and providers have witnessed great advances in the treatment of this disease. With these advances, there has been a dramatic decrease in the mortality and morbidity associated with hemophilia. Even with the remarkable advancements in treatment, however, new and old challenges continue to plague the hemophilia community. The cost of factor replacement and the frequency of infusions, especially in patients with severe hemophilia on prophylaxis, remains a significant challenge for this population. Other challenges include obtaining reliable venous access, especially in younger patients, and the development of neutralizing alloantibodies (inhibitors). The development of extended half-life products, a bispecific antibody which mimics the coagulation function of factor VIII (FVIII) and inhibition of anticoagulation proteins such as antithrombin with antibodies, aptamers or RNA interference technology have offered novel therapeutic approaches to overcome some of these existing challenges. Additionally, ongoing gene therapy research offers a way to possibly cure hemophilia. These novel treatment tools in conjunction with the establishment of an increasing number of comprehensive hemophilia centers and worldwide advocacy efforts have continued to push the progress of hemophilia care to new frontiers. This review highlights and summarizes these novel therapeutic approaches and the current clinical progress of hemophilia A.

Social support and resilience in persons with severe haemophilia: An interpretative phenomenological analysis

“…Inhibitors present a serious challenge to persons with haemophilia (PWH): their presence makes treatment less effective than the treatment of haemophilia without inhibitors, and bleeds tend to be significantly more frequent 7 . Although new treatment options for both inhibitor and non‐inhibitor patients, including those with subcutaneous application, are emerging on the market, 8 most patients are still treated with repeated intravenous infusions. This places additional burden on patients whose quality of life is already compromised 9 .…”

Patient Perspectives on Novel Treatments in Haemophilia: A Qualitative Study

“…Some of these products may be administered subcutaneously and no longer require intravenous injections. However, new treatments may have drawbacks of their own, including known and unknown risks, as summarised in Table 1 [10][11][12][13][14].…”

“…Overview of haemophilia A and B treatment products currently under development or marketed recently, with their benefits, potential disadvantages and mechanisms of action[10][11][12][13][14] …”

Tolerance induction in hemophilia: innovation and accomplishments

“…Clotting factor replacement products can be used prophylactically or "on demand" in trauma or surgical intervention. Factor products have a relatively short half-life and require frequent administration [11,12,13]. One approach to increase systemic retention of clotting factors is pegylation.…”