The French haemophilia cohort: rationale and organization of a long-term national pharmacosurveillance system.

Medicinal products of biological origin still carry a specific iatrogenic risk, mainly because of their starting material, mode of preparation and variability. Careful postmarketing surveillance systems are therefore necessary. To assess the long-term safety of haemophilia treatment with plasma-derived and recombinant clotting factor products, a cohort study was set up in France in 1994. Participants were patients with haemophilia A and B, with or without previous clotting factor therapy. Clinical events, treatments, biological data and adverse events were recorded on standard forms. Blood samples were separated into serum, plasma and peripheral blood mononuclear cells, frozen, and banked in a central laboratory. The same data and samples were collected at yearly follow-up visits. As of December 1999, 1234 haemophiliacs were enrolled in 39 haemophilia centres. At enrollment, 50.2% of patients were under 15 years of age, and the cumulative number of days of exposure to the product was below 50 in 35.1% of cases. The median duration of follow-up was 26.9 months, with a total of 2729 patient-years (135,947 days of exposure and 211 million units of factor VIII or IX). To date, only 17 patients were lost to follow-up. The initial results show good compliance with this health-watch policy among patients and clinicians specializing in haemophilia. The regular follow-up data and centralized sample bank will serve to investigate rapidly any suspected outbreaks as soon as reliable biological tests become available in the future.