The COST-UTILITY ANALYSIS OF FOUR CHELATION REGIMENS FOR Β-THALASSEMIA MAJOR: A CHINESE PERSPECTIVE

Abstract

Background: The four most commonly used chelation regimens for β-thalassemia major patients in China are a combination therapy of deferoxamine and deferiprone (DFO+DFP), deferoxamine(DFO) monotherapy, deferiprone(DFP) monotherapy and deferasirox(DFX) monotherapy. Such patients use iron chelators their whole lives, resulting in enormous treatment costs. This study analyses the cost-utility of these four regimens from the Chinese healthcare system perspective.
 Methods: A Markov decision model was used over a 70-year time horizon and was populated using clinical data from a systematic literature review. We obtained utility data from local and previous research. Costs were estimated using Chinese national sources.
 Results: From the base-case analysis results, DFP was the most cost-effective chelation regimen, followed by DFO+DFP, DFO and DFX. DFP had a 99.60%, 78.10% and 64.40% likelihood of being cost-effective versus DFX, DFO and DFO+DFP, respectively, at a payment threshold of 193,932.00 CNY/QALY.
 Conclusions: DFP was the most cost-effective chelation regimen for β-thalassemia major patients, followed by DFO+DFP, DFO and DFX. Using DFP as the primary treatment regimen may potentially result in cost-savings and QALY gains for the Chinese healthcare system. To increase these benefits, the Chinese government and clinicians should lower drug costs, increase drug utility and reduce mortality and morbidity. Changes in influential parameters easily affect the results of DFO+DFP versus DFP and of DFP versus DFO; clinicians should focus on such parameters and adjust the regimens accordingly.

New Entity—Thalassemic Endocrine Disease: Major Beta-Thalassemia and Endocrine Involvement

“…It is categorised into three main subtypes: major, intermedia and minor. Traditionally, BTH is highly prevalent in Mediterranean, Middle Eastern, and Southeast Asian countries; modern dynamics of the population increased the prevalence in North America, as well as Central and North Europe [ 1 , 2 , 3 , 4 , 5 , 6 , 7 , 8 , 9 , 10 ].…”

“…Worldwide, incidence is 0.3–0.4 million persons/year who are born with different types of haemoglobinopathies, thalassemia representing the most frequent hereditary anomaly of haemoglobin [ 1 , 2 , 3 , 4 , 5 , 6 , 7 , 8 , 9 , 10 , 11 ]. The damage of erythropoiesis induces premature erythrocytes destruction, causing chronic anaemia with extra-medullary haematopoiesis (EH) and associated bone marrow expansion (BME) in TH individuals (individuals diagnosed with major beta-thalassemia) [ 1 , 2 , 3 , 4 , 5 , 6 , 7 , 8 , 9 , 10 ].…”

“…Worldwide, incidence is 0.3–0.4 million persons/year who are born with different types of haemoglobinopathies, thalassemia representing the most frequent hereditary anomaly of haemoglobin [ 1 , 2 , 3 , 4 , 5 , 6 , 7 , 8 , 9 , 10 , 11 ]. The damage of erythropoiesis induces premature erythrocytes destruction, causing chronic anaemia with extra-medullary haematopoiesis (EH) and associated bone marrow expansion (BME) in TH individuals (individuals diagnosed with major beta-thalassemia) [ 1 , 2 , 3 , 4 , 5 , 6 , 7 , 8 , 9 , 10 ]. Blood profile typically includes microcytic anaemia, and high or normal ferritin; Hb electrophoresis highlights the subtype of TH, and genetic testing confirms the disease, included as part of the prenatal protocol [ 1 , 2 , 3 , 4 , 5 , 6 , 7 , 8 , 9 , 10 ].…”

“…TH carriers are usually asymptomatic; major alpha TH is associated with a very high mortality at birth. In order to survive, patients with major BTH require lifelong transfusions beginning in early childhood; intermediate alpha and beta subtypes have heterogeneous presentations from severe to mild forms [ 1 , 2 , 3 , 4 , 5 , 6 , 7 , 8 , 9 , 10 ]. BTH-related morbidities are caused by EH, BME, and iron overload (IO) in the endocrine glands, skeleton, heart, liver, pancreas, and kidneys, etc.…”

“…BTH-related morbidities are caused by EH, BME, and iron overload (IO) in the endocrine glands, skeleton, heart, liver, pancreas, and kidneys, etc. [ 1 , 2 , 3 , 4 , 5 , 6 , 7 , 8 , 9 , 10 ]. IO induces an excessive amount of reactive oxygen species (ROS) with consequent organ and vascular damage [ 1 , 2 , 3 , 4 , 5 , 6 , 7 , 8 , 9 , 10 , 11 ].…”