Hemophilia: an amazing 35-year journey from the depths of HIV to the threshold of cure

Abstract

Methods developed in the early 1970s to highly purify factor VIII (FVIII) from the plasma of large numbers of blood donors led, for the first time, to concentrates of FVIII that enabled hemophiliac to self-treat, providing independence and opening the way to safe surgery and other treatments. But, with the introduction of blood-borne viruses such as HIV-1 and hepatitis C viruses into the blood supply, these concentrates also transmitted HIV and hepatitis to a high percentage of hemophiliacs. Nevertheless, from the depths of the AIDS epidemic in hemophilia came extraordinary scientific advances that led to recombinant FVIII, the identification of HIV as the agent causing AIDS, the eventual development of effective treatments for AIDS, gene transfer approaches using lentiviruses, and treatments for hepatitis C. All of these have improved the lives of current and future hemophiliacs and have brought us to the threshold of a cure.

Fig. 1

AIDS and Hemophilia 1977–2002. The graphs show national numbers for yearly and cumulative infection with HIV-1 and death in hemophilia. Front row bars, number of individuals infected by year; second row bars, number of deaths due to AIDS by year; third row bars, cumulative infections by year; last row bars, cumulative deaths by year. There were 3915 deaths in hemophilia nationwide at the time of this analysis; 135 hemophiliacs died of AIDS at the UNC Center. The names in the background were the 135 patients who died at the UNC Center, but have been changed to John Doe to comply with HIPAA regulations. (copyright G. C. White, II).

Fig. 2

Initial Vials of rFVIII infused to a Hemophiliac. The infusion set for the first administration of rFVIII to a human on March 27, 1987 is shown on the left. The vial containing the first clinical lot (#2905E001AA1) of rFVIII manufactured on March 17, 1987 is shown on the right (copyright G. C. White, II).