Abstract
Chronic infection with the hepatitis C virus (HCV) has long been the dominant complication
of substitution therapy in patients with inherited blood disorders and the cause of
anticipated death due to end-stage liver disease. In hemophilia, transmission of HCV
with clotting factors concentrates started to be curbed in the mid-1980s following
the adoption of procedures of virus inactivation of concentrates based on heat, whereas
in the 1990s treatment of HCV infection with interferon monotherapy was attempted,
however, with little success. The advent of combination therapy of interferon with
ribavirin led to a substantial improvement of treatment outcome (40% rate of cure),
that however was still of limited efficacy in patients with advanced liver disease,
those with high load of HCV genotype 1, and patients coinfected with the human immunodeficiency
virus. In this latter population, while the course of hepatitis C was accelerated
as a consequence of immunodeficiency, the advent of highly active antiretroviral therapy
led acquired immunodeficiency syndrome (AIDS) to decline and hepatitis C to progressively
emerge as a dominant cause of mortality, in parallel. In patients with thalassemia,
transfusion-related transmission of HCV was efficiently interrupted in 1992 with the
advent of sensitive screening tests for testing donors for HCV, whereas treatment
with interferon and ribavirin of infected thalassemics was constrained by an increased
risk of anemia due to the hemolytic properties of ribavirin coupled with interferon-induced
bone marrow suppression. The advent of safe and potent regimens based on the oral
administration of direct antiviral agents has revolutionized therapy of HCV in patients
with congenital blood diseases, providing substantial clinical benefits and making
elimination of infection in these populations, possible.
Keywords
hepatitis C - hemophilia - sickle cell anemia - thalassemia - direct antiviral agents
