Factor concentrate usage in persons with hemophilia in New York State.

Background: Persons with a congenital deficiency of FVIII or F IX (hemophilia A and hemophilia B, respectively) receive factor concentrate to treat or prevent bleeding.

Study Design And Methods: A population-based study of all persons with hemophilia residing in New York State at any time during 1993 through 1998 was conducted. All available medical records for each patient were reviewed to determine type of therapy.

Results: Case finding yielded 1160 cases, for a prevalence of 63.9 per 1 million population in 1998. Recombinant factor concentrates were used by 56 percent of patients. Patients with severe disease used more (158,234 IU/patient) factor concentrate than did patients with moderate disease (46,315 IU) or mild disease (5794 IU). Over half (57%) of all factor concentrate was prescribed for patients with severe disease on prophylactic therapy. Patients undergoing immune tolerance therapy used the most per person-455,116 IU each. Hemophilia treatment centers provided factor concentrate for 62 percent of all patients who used factor and 73 percent of patients with severe disease.

Conclusion: Hemophilia patients, especially patients with severe disease, use large amounts of expensive factor concentrates to prevent and to treat bleeding episodes. Specialized hemophilia treatment centers play a key role in the care of these patients.