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The Cost-Effectiveness of Alternative Iron Chelating Agents for the Treatment of Transfusional Hemosiderosis in Patients with Sickle Cell Disease
Başlık:
The Cost-Effectiveness of Alternative Iron Chelating Agents for the Treatment of Transfusional Hemosiderosis in Patients with Sickle Cell Disease
Yazar:
Brown-Bickerstaff, Cherrishe, author.
ISBN:
9780438009202
Yazar Ek Girişi:
Fiziksel Tanımlama:
1 electronic resource (85 pages)
Genel Not:
Source: Dissertation Abstracts International, Volume: 79-10(E), Section: B.
Advisors: Vakaramoko Diaby Committee members: Ellen Campbell; Matthew Dutton; Thomas Harrington; Arinzechukwu Okere.
Özet:
Sickle cell disease is the most common hemoglobinopathy in the U.S. Frequent blood transfusions are often used to treat and prevent disease related complications in this population. Unfortunately, chronic blood transfusions can result in transfusional hemosiderosis, an excess accumulation of non-transferrin bound iron which can deposit in bodily tissues causing damage to organs such as the liver and the heart. Iron chelating therapies deferoxamine and deferasirox are two FDA approved therapies that aid in the removal of excess iron from the body due to chronic blood transfusions.
The research aim of this dissertation was to thoroughly assess the cost-effectiveness of alternative iron chelating agents for the treatment of transfusional hemosiderosis in patients with sickle cell disease. To do so, a systematic review of cost-effectiveness studies of treatment alternatives for iron overload in patients with thalassemia and sickle cell disease was carried-out. Studies were identified through a literature search of health and economic databases and were independently checked against inclusion and exclusion criteria. Available evidence indicated the cost-effectiveness of deferasirox and deferiprone in beta thalassemia patients. There was little to no information on the cost-effectiveness of iron chelating therapies among sickle cell disease patients.
A comprehensive assessment of the cost-effectiveness of available iron chelating therapies for the treatment of transfusional hemosiderosis in U.S. patients with sickle cell disease was conducted. A Markov model was developed to mimic the disease progression of sickle cell disease patients with chronic iron overload in deferasirox and deferoxamine treatment arms. This model was used to estimate the associated costs and health utilities for each treatment arm. While the total costs of deferasirox exceeded those of deferoxamine, deferasirox therapy was also associated with greater health utility. As such, deferasirox was found to be cost-effective with an estimated incremental cost-effectiveness ratio of 26,025.20 per QALY gained. Sensitivity analyses did not alter the direction of the findings of this study.
Finally, this dissertation assessed the financial impact from the U.S. Medicaid perspective of increasing the market share of deferasirox therapy in the iron-overloaded sickle cell disease population. While deferasirox was shown to increase cost and have a significant budget impact, this was driven by the lack of compliance, and thus reduced costs associated with deferoxamine use.
Notlar:
School code: 0872
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Yer Numarası | Demirbaş Numarası | Shelf Location | Lokasyon / Statüsü / İade Tarihi |
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