Haemophilia: provision of factors and novel therapies: World Federation of Hemophilia goals and achievements

Abstract

Summary For nearly 50 years, the goal of the World Federation of Hemophilia (WFH) has been to achieve ‘Treatment for All’ patients with inherited bleeding disorders, regardless of where they live. With proper diagnosis, management and care, people with bleeding disorders can live perfectly healthy lives. Without treatment, the reality is that many will die young or, if they survive, suffer joint damage that leaves them with permanent disabilities. Only about 25% of the estimated 400 000 people with haemophilia worldwide receive adequate treatment. The percentage is far lower for those with von Willebrand Disease (VWD) and the rarer bleeding disorders. The achievements of the WFH to close the gap in care for people with bleeding disorders are measureable over time by using three key indicators; the difference in the estimated and actual number of people known with bleeding disorders, the amount of treatment products needed versus that available, and the number of people born with bleeding disorders and the number who reach adulthood. There are five essential elements to achieve a sustainable national care programme: ensuring accurate laboratory diagnosis, achieving government support, improving the care delivery system, increasing the availability of treatment products, and building a strong national patient organization.

Haemophilia Experiences, Results and Opportunities (HERO study) in Brazil: Assessment of the psychosocial effects of haemophilia in patients and caregivers

“…However, the exact numbers may be higher, as only approximately one third of the people with this condition have been diagnosed . Unfortunately, most patients with severe haemophilia have limited access to adequate treatment, as they live primarily in developing countries . Without adequate treatment, haemophilia can be extremely debilitating and can present a significant economic burden on patients, caregivers and society, influencing education and employment .…”

Thrombin generation assay using factor XIa to measure factors VIII and IX and their glycoPEGylated derivatives is robust and sensitive

“…Hemophilia is a congenital bleeding disorder characterized by the deficiency of coagulation factor VIII (FVIII) in hemophilia A (HA) or factor IX (FIX) in hemophilia B (HB). Hemophilia affects an estimated 400 000 individuals worldwide , with HA representing 80–85% of the total hemophilia population . Depending on the level of coagulation factor deficiency, hemophilia is classified as mild, moderate, or severe (> 5 to < 40%, 1–5% and < 1% of normal factor activity, respectively) .…”

Ensuring maximum outcomes and benefits in comprehensive care for bleeding disorders through surveillance and data collection

“…5 The improved outcomes in morbidity and mortality when comprehensive care occurs within an HTC setting are well established. 6 In low resourced countries where treatment is typically not immediately and consistently available, patients are at increased risk of suffering severe and permanent disability and early death. 7…”