Outcomes, utilization, and costs among ... - Wiley Online Library

Outcomes, utilization, and costs among thalassemia and sickle cell disease patients receiving deferoxamine therapy in the United States Thomas E. Delea,1* May Hagiwara,1 Simu K. Thomas,2 Jean-Francois Baladi,2 Pradyumna D. Phatak,3 and Thomas D. Coates4 Deferoxamine mesylate (DFO) reduces morbidity and mortality associated with transfusional iron overload. Data on the utilization and costs of care among U.S. patients receiving DFO in typical clinical practice are limited however. This was a retrospective study using a large U.S. health insurance claims database spanning 1/97–12/04 and representing 40 million members in >70 health plans. Study subjects (n 5 145 total, 106 sickle cell disease [SCD], 39 thalassemia) included members with a diagnosis of thalassemia or SCD, one or more transfusions (whole blood or red blood cells), and one or more claims for DFO. Mean transfusion episodes were 12 per year. Estimated mean DFO use was 307 g/year. Central venous access devices were required by 20% of patients. Cardiac disease was observed in 16% of patients. Mean total medical costs were $59,233 per year including $10,899 for DFO and $8,722 for administration of chelation therapy. In multivariate analyses, potential complications of iron overload were associated with significantly higher medical care costs. In typical clinical practice, use of DFO in patients with thalassemia and SCD receiving transfusions is low. Administration costs represent a large proportion of the cost of chelation therapy. Potential complications of iron C 2007 Wiley-Liss, Inc. overload are associated with increased costs. Am. J. Hematol. 83:263–270, 2008. V

Introduction Patients with thalassemia and sickle-cell disease (SCD) frequently require chronic transfusion therapy. Humans cannot actively eliminate iron from the body, however, and such therapy results in an accumulation of iron in the heart, spleen, liver, and endocrine organs. Untreated, these accumulations may result in a host of ultimately fatal complications, including cardiac disease, diabetes, hypogonadism, hypoparathyroidism, hypothyroidism, and liver disease [1]. Deferoxamine mesylate (DFO) is an iron chelator that has been shown to reduce iron-related morbidity and mortality in patients with transfusion-dependent thalassemia [2– 5]. However, the poor bioavailability and short plasma halflife of DFO require that it be administered as a slow subcutaneous or intravenous infusion over an 8–12 h period 5– 7 times per week. The cumbersome nature of this regimen may lead to poor compliance in many patients. Retrospective analyses of patients with b-thalassemia receiving DFO have found that those with good compliance (>250 infusions per year) have favorable prognosis, with approximately 90% survival to age 30 years, whereas prognosis for those with poor compliance (