Abstract
Background: Patients suffering from β-thalassemia or sickle cell disease require on-going blood transfusions. Chronic transfusion, however, results in iron overload, which if not removed by iron chelation therapy (ICT), causes organ damage. Deferoxamine (DFO) is currently the standard of care for ICT, but many patients do not adhere to therapy possibly because of the need for almost daily infusions lasting 8 to 10 hours each.
Rationale: While the impact of current care on clinical and patient outcomes is generally understood, less is known about the total cost of DFO therapy.
Objectives: To identify a complete set of cost items to inform the development of an ICT related Resource Use Questionnaire (RUQ) for administration in an international cohort study of the actual cost of ICT in practice; and to obtain a preliminary, literature-based estimate of total annual per patient costs of ICT.
Methods: A search of the literature (EMB Reviews; Scirus and Ovid Medline (1996+); PubMed (1995+) was performed using the following key words: thalassemia, sickle cell disease, myelodysplastic syndrome, cost, iron chelation, Desferal, deferoxamine, resource use, reimbursement and compliance. Cost items were extracted from eligible studies to create an aggregated, composite set of ICT-related variables to which unit costs (2004/2005 USD) were applied.
Results: Of 396 abstracts obtained, all but 96 were excluded because ICT cost data were lacking. Of those retained, only 4 studies (1 Israël;1 US;2 UK) reported ICT-related costs (1 lifetime;3 annual). Cost variables differed markedly among studies each focusing on some specific aspect. The application of unit costs to the composite list of ICT-related variables and associated resource use profiles reveal that total annual per patient ICT costs may be as high as $7,487 to $15,836 (£4,191 to £8,865) depending on age. The cost of DFO accounts for only 16%–31% of these estimated total costs, with the balance accounted for by other annual ancillary expenditures such as equipment and supplies, monitoring, and home health care services. Total costs could well be underestimated given that component lifetime costs such as DFO treatment complications, the clinical sequelae of poor adherence to DFO, and the indirect costs of lost productivity were not included. Cost estimates will be supplemented and validated at the time of abstract presentation by the resource use and unit cost data generated by the RUQ employed in the aforementioned international cohort study.
Conclusions: Estimated total costs of ICT are substantial and well exceed the cost of DFO alone. A paucity of published data related to the total costs of ICT underscores the need for additional ICT cost data from actual practice to better understand the economic impact of novel ICT agents.
