Clinical and economic burden of infused iron chelation therapy in the United States

BACKGROUND: Patients requiring chronic blood transfusions are at risk for iron overload, which, if not treated by iron chelation therapy (ICT), can create serious organ damage and reduce life expectancy. Current ICT requires burdensome 8‐ to 12‐hour infusions five to seven times per week. STUDY DESIGN AND METHODS: A naturalistic study of the burden of infused ICT was conducted in four US centers. Data from the initial and most recent years of ICT were collected from medical charts of consenting thalassemia (n = 40) and sickle cell disease (n = 9) patients. Quality of life (QoL), treatment satisfaction, and ICT‐related resource utilization data were also collected from a patient interview. RESULTS: Mean serum ferritin levels during the initial (2519 ± 1382 ng/mL) and most recent (2741 ± 2532 ng/mL) years remained unacceptably high and increased over time (306 ± 2200 ng/mL; mean of 20± years of therapy). Within 30 days before interview, 55 percent of patients suffered at least one ICT‐related adverse event; 76 percent missed at least one dose. QoL, measured by the SF‐36, and treatment satisfaction appear compromised in this cohort. Although total annual costs of ICT were estimated at USD $30,000 to $35,000, drug accounted for only 50 to 60 percent of this amount. CONCLUSIONS: Infused ICT may not provide adequate effectiveness in the real world. High ferritin levels seem to be associated with ICT noncompliance, likely in relation to the bothersome mode of administration and side effects. The total cost of ICT appears to well exceed that of drug alone.