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Jialian Li


β-thalassemia major Deferoxamine, Deferiprone, Deferasirox, cost-effectiveness, systematic review


Deferoxamine (DFO) or Deferiprone (DFP) or Deferasirox (DFX) monotherapy and DFO and DFP combination therapy were four commonly implemented now chelation regimens for the iron overloaded of ?-thalassemia major. This systematic review aims to determine the cost-effectiveness of four chelation regimens and provide evidence for the rational use of chelation regimens for ?-thalassemia major therapy in clinic.
A systematic literature search in PubMed, EMBASE (Ovid), CENTRAL (Cochrane library), HTAD (Cochrane library), NHS EED (Cochrane library), CBM, CNKI, VIP, and Wanfang was conducted in April 2018. In addition, a manual search was performed. Two researchers, working independently, selected the papers, extracted the data and assessed the methodological quality of the included papers. Each included paper was evaluated using a checklist developed by Drummond et al.
The initial number of records was 968, and eight papers met the final eligibility criteria. All the included eight papers were cost-utility analyses. And the methodological quality of these papers was good. Nineteen studies were included in eight papers. Nine studies of DFX versus DFO had contradictory results. Out of the nineteen studies, three studies of DFX versus DFP established that using DFP was cost-effective. Three studies of DFP versus DFO established that using DFP was cost-effective. One study of DFP and DFO combination therapy versus DFO found that using DFO was cost-effective. One study of DFP and DFO combination therapy versus DFP found that using DFP was cost-effective. And there were two studies of DFP and DFO combination therapy versus DFX, but we cannot be sure which one of two chelation regimens was cost-effective.
In brief, DFP is the best choice, followed by DFO or DFX, when an iron chelator is to be used alone for ?-thalassemia major therapy. All studies that compared DFO and DFP combination therapy with DFO (or DFP or DFX) monotherapy established that the combination therapy with DFO and DFP was not cost-effective. However, due to the low number of related studies, more extensive, high-quality research is required for further analysis and confirmation of our findings. Moreover, the cost effectiveness is not an absolute issue when in different countries(regions) the results are opposite for other countries(regions). The specific region had a substantial influence on the economy of drugs.

Key words: ?-thalassemia major, Deferoxamine, Deferiprone, Deferasirox, cost-effectiveness, systematic review


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