Inherited bleeding disorders are caused by the deficiency or dysfunction of plasma proteins required for the development of a physiological hemostatic process1,2. Von Willebrand disease (vWD), haemophilia A and haemophilia B are the most common inherited disorders of hemostasis. In their severe forms, these disorders are life-threatening. However advanced treatment modalities have enabled even severely affected patients to reach the same life expectancy of the general male population at least in high-income countries3,4. Indeed, after the dramatic events of widespread blood-borne virus transmission in the 1970s–1980s in haemophilic patients, leading to a 35-to-40-year reduction in life expectancy in Canadian patients5, there has been a strong drive towards continuous improvement, primarily in the safety of replacement therapy6. There is now a very high degree of safety from risk of blood borne transmission of pathogens as evidenced by the absence of reported transmission of blood-borne viruses in persons with haemophilia since the late 1980s to date7. The safety of current replacement therapies was recently confirmed by a prospective surveillance program ongoing since 2008 and based on regular monitoring of 22,242 European patients (European Haemophilia Safety Surveillance System; www.euhass.org). Regular factor prophylaxis as a therapeutic regimen has been proven effective in preventing haemophilic arthropathy8. Even in the presence of neutralizing antibodies (inhibitors) directed against the deficient clotting factor, particularly in haemophilia A, bleeding can usually be treated successfully using activated variants of coagulation factors9. Moreover, immune tolerance induction (ITI), based on the long-term intravenous infusion of large doses of FVIII, eradicates inhibitors in as many as two-thirds of patients10. Therefore, improving quality of life has become the primary objective of care in developed countries; thus multiple modalities (psychosocial support, physiotherapy, integration into community life, etc), not just the infusion of the deficient factor, are made available to the patient and his family, to allow them to fully experience good health.
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