Utilization and Costs of Deferoxamine in Patients with Thalassemia, Sickle-Cell Disease, or Myelodysplastic Syndrome Receiving Frequent Transfusions.
Authors: Delea Thomas E. , Hagiwara May , Thomas Simu K. , Baladi Jean-Francois , Phatak Pradyumna D. , Coates Thomas D. 
Published: April 2020

Background. Patients with thalassemia, sickle-cell disease (SCD), and myelodysplastic syndromes (MDS) receiving frequent transfusions require chelation therapy to prevent complications of iron overload. Deferoxamine (DFO) is an effective iron chelator that has been shown to reduce the morbidity and mortality associated with transfusional hemosiderosis. Data on the utilization and costs of DFO treatment are limited however. The objective of this study was to document the utilization and costs of DFO therapy in patients with transfusion-dependent anemias seen in typical clinical practice.

Methods. Retrospective, observational study using data from large health insurance claims database spanning 1/97–12/04 (“study period”) and representing approximately 40 million members enrolled in &gt;70 health plans across the US. Study subjects included members meeting the following criteria:

≥1 claims with diagnosis of thalassemia (282.4x), SCD (282.6x ), or MDS (ICD-9-CM 238.7x); ≥8 claims (on different days) for a transfusion of whole blood or red cells; ≥2 claims (on different days) for DFO.

Follow-up was defined as the period from the date of first DFO claim (“index date”) to end of study period, disenrollment, or 15 days after last claim for DFO, whichever occurred first. Outcomes included the number of claims for DFO and grams of DFO dispensed and the costs of DFO therapy, including costs of drug acquisition and administration. Outcomes were analyzed by qualifying diagnosis, numbers of transfusions received, and grams of DFO dispensed.

Results. We identified 155 subjects who met all inclusion criteria, including 35 with thalassemia, 68 with SCD, and 52 with MDS. On average, patients received one transfusion every 3.4 weeks of follow-up. Mean DFO grams dispensed were 306 per year. MDS patients received the most transfusions but the least DFO. Only 38% of MDS patients received ≥3 g of DFO per week (≥156 g per year). Mean total DFO costs were $18,025 annually ($10,217 for drug and $7,808 for administration). Controlling for other factors, utilization of DFO was not associated with number of transfusions received; administration costs were only weakly associated with amount of DFO received.

Thalassemia SCD MDS All Values are Mean±SD N 35 68 52 155 Follow-up, days 612 ± 481 420 ± 403 274 ± 336 414 ± 418 Age, years 19 ± 12 17 ± 11 63 ± 11 33 ± 24 Transfusions per year 15 ± 7 12 ± 4 24 ± 13 16 ± 10 DFO claims per year 29 ± 34 41 ± 46 30 ± 20 34 ± 37 DFO grams per year 311 ± 233 343 ± 243 223 ± 234 306 ± 241 Cost DFO acquisition, $ per year 10,287 ± 8,264 11,625 ± 8,339 7,293 ± 7,543 10,217 ± 8,207 Cost DFO administration, $ per year 7,674 ± 11,503 9,109 ± 8,177 5,403 ± 5,649 7808 ± 8,438 Total cost of DFO, $ per year 17,961 ± 17,047 20,734 ± 12,114 12,696 ± 10,886 18,025 ± 13,348

Conclusion: In this population of frequently transfused patients (mean 16 transfusions per year), utilization of DFO was low (mean &lt;1 gram per day) suggesting inadequate chelation. Costs of DFO administration were high, representing approximately 43% of the total cost of chelation.