Back to the future: a recent history of haemophilia treatment

Abstract

Summary. In the last few decades, the management of patients with haemophilia has witnessed dramatic improvements, through the larger availability of safe plasma-derived and recombinant products for replacement therapy. Another important step forward is the progressively larger-scale implementation of primary prophylaxis in children. Currently, the main problem in patients with haemophilia is the onset of antibodies inactivating the infused clotting factor (inhibitors), even though immune tolerance regimens that are able to eradicate inhibitors and the availability of products that bypass the intrinsic coagulation defects have dramatically improved the management of these patients. Cure of haemophilia through gene transfer is being attempted, but relatively, it is far from being implemented on a large scale. It is likely that further improvements in replacement therapy will occur in the near future, through the availability of new-therapeutic tools such as factors VIII and IX with longer half-lives, more potent bypassing agents and factors extracted from the milk of transgenic animals.

Optimizing management of immune tolerance induction in patients with severe haemophilia A and inhibitors: towards evidence‐based approaches

“…Thanks to the large availability of safe factor VIII (FVIII) concentrates for replacement therapy and the diffusion of primary prophylaxis to prevent arthropathy, at least in highincome countries, haemophilia A patients currently receive excellent treatment and achieve a satisfactory quality of life (Mannucci, 2008). Nevertheless, the development of antibodies against therapeutically administered FVIII (inhibitors) remains the most serious complication of treatment of haemophilia A. Inhibitors occur in approximately 30% of severely affected [FVIII coagulant activity (FVIII:C) <1%] previously untreated patients (PUPs), usually during the first 50 exposure days, as a result of interactions between multiple genetic and environmental factors .…”

How I treat age-related morbidities in elderly persons with hemophilia

“…1 Because of the increasing availability of coagulation factor concentrates (CFCs), at least in high-income countries, life expectancy increased in persons with hemophilia (PWHs) from less than 30 years to more than 60 years. [2][3][4][5][6][7] This favorable trend was temporarily halted in the last 2 decades of the 20th century by the devastating impact of infections with the human immunodeficiency virus (HIV) and the hepatitis C virus (HCV).…”

The Hemophilic Dental Patient: A Case Report

“…Recurrent joint bleeds inevitably leading to crippling arthropathy (Rosendaal and Lafeber, 2006), were the hallmarks of the disease before the 1970s, when plasma fractions containing FVIII or FIX were still not available. At that time mortality from bleeding was high, and the life expectancy of persons with hemophilia was much lower than that of general population (Munnucci, 2008;Plug et al, 2004).…”