Effectiveness Of Deferasirox Research Articles

Effectiveness of Deferasirox in Pediatric Thalassemia Patients: Experience from a Tertiary Care Hospital of Odisha.

BACKGROUND AND OBJECTIVES:Patients with beta-thalassemia require lifelong blood transfusions, leading to chronic iron overload, which can lead to growth retardation, as well as hinder sexual development during the adolescent period and dysfunction of organs such as heart, pancreas, and endocrine glands. These patients are in need of lifelong transfusion therapy and hence lifelong iron chelation therapy as well. Hence, this study was aimed to assess the effectiveness of deferasirox for iron chelation in pediatric thalassemia cases in a tertiary care hospital of Eastern India.SUBJECTS AND METHODS:This prospective, observational, hospital-based study was conducted from June 2015 to December 2016. Two hundred and fifty patients were assessed for eligibility, of which 174 were included. Effectiveness of deferasirox was observed by measuring serum ferritin levels which were monitored at the end of every 3 months till 1 year. We also evaluated the compliance with deferasirox therapy in the same study cohort.RESULTS:The serum ferritin level reduced significantly at the end of 12 months in comparison to baseline (P = 0.04). There was a mean absolute decrease in serum ferritin only in the dose range of 21–30 mg/kg/day. Approximately 90% of the patients had 100% compliance with deferasirox therapy.CONCLUSIONS:Deferasirox is an effective iron chelator when started at an optimum time and with optimum dose. At least 1 year of deferasirox therapy is needed for a significant lowering of serum ferritin levels of pediatric thalassemia patients on multiple blood transfusions.

Cost-effectiveness Analysis of Deferiprone and Deferasirox on Thalassemia Major Patients in Tangerang District Hospital, Indonesia

Background: Blood transfusion are needed in improving the quality of life of thalassemia major patients. However since it can lead to excess iron, the iron chelation therapy is needed. Deferiprone and deferasirox are the most often used therapy in Indonesia. Objective: The aim of this study was to compare the cost-effectiveness of deferasirox with deferiprone with costeffectiveness analysis (CEA). Methods: Data were taken retrospectively and sampling was done using total sampling based on medical records and hospital information system. Serum ferritin levels of patients consuming deferasirox (n=27) and deferiprone (n=33) were measured to observe the mean changes of serum ferritin levels as effectiveness’ parameter. The cost was median of the total direct medical cost, summed from the cost of drugs, medical devices, hospitalization, administration, physician, laboratories and blood bags. Results: Based on the results of this study, the effectiveness of deferasirox (1164 ng/mL) was greater than deferiprone (692 ng/mL). Median total cost of deferasirox was more expensive (Rp 76,610,618.69) than deferiprone (Rp 51,869,965.64). Cost-effectiveness ratio of deferasirox (CER: Rp 65,816.68/effectiveness) was lower than deferiprone (CER: Rp 74,956.60/ effectiveness). None of both medications was dominant and therefore we could not determine which medication was the most cost-effective therapy. Changing of medication from deferiprone to deferasirox requires an extra cost of Rp 52,416.64 per one incremental unit of effectivity. Conclusion : The policy maker in healthcare facility need to consider the budget and whether the incremental cost of deferasirox is proportional to its increased effectiveness. Key words: Cost-effectiveness analysis, deferiprone, deferasirox, iron chelation therapy, thalassemia major.

Observation on the clinical effectiveness of deferasirox, deferiprone and desferrioxamine in the treatment of iron load in patients with thalassemia major

Objective To explore the clinical effectiveness of deferasirox, deferiprone and desferrioxamine in the treatment of iron overload in patients with thalassemia major. Methods Among the severe thalassemia patients with iron overload being diagnosis, treated and followed up in Guigang City People′s Hospital, fifty cases were tested by MRI T2 and were detected with cardiac and liver iron overload, the patients were selected and randomly divided into the deferoxamine treatment (DFO) group(18 case), deferiprone treatment (DFP) group (22 cases) and deferasirox treatment (DFX) group (10 case), The baseline data of the three groups were statistically analyzed.the serum ferritin (SF) value, the cardiac MRI T2 value and the liver MRI T2 value before and after treatment were measured. Results The patients completed the treatment, after 1 years of treatment, the serum SF value, cardiac and liver MRI T2 values of the patients were improved than those before the treatment (P 0.05); the differences in liver MRI T2 value among the three groups were not statistically significant(F=0.131, P>0.05), There were significant differences in heart iron load MRI T2 values among the three groups ((34.27 ± 6.30) ms vs.(23 ± 2.79) ms vs.(21.8 ± 2.20)ms, F=39.54, P<0.05). Conclusion Deferoxamine, deferiprone and deferasirox can effectively alleviate the iron load in patients with thalassemia major.in terms of cardiac iron overload, the curative effects of deferiprone treatment in patients with thalassemia is superior than desferrioxamine and deferasirox treatment, it is worthy of clinical promotion. Key words: Thalassemia Major; Iron Chelator; Serum Ferritin; MRI T2*Ttechnology

Korean Guideline for Iron Chelation Therapy in Transfusion-Induced Iron Overload

Many Korean patients with transfusion-induced iron overload experience serious clinical sequelae, including organ damage, and require lifelong chelation therapy. However, due to a lack of compliance and/or unavailability of an appropriate chelator, most patients have not been treated effectively. Deferasirox (DFX), a once-daily oral iron chelator for both adult and pediatric patients with transfusion-induced iron overload, is now available in Korea. The effectiveness of deferasirox in reducing or maintaining body iron has been demonstrated in many studies of patients with a variety of transfusion-induced anemias such as myelodysplastic syndromes, aplastic anemia, and other chronic anemias. The recommended initial daily dose of DFX is 20 mg/kg body weight, taken on an empty stomach at least 30 min before food and serum ferritin levels should be maintained below 1000 ng/mL. To optimize the management of transfusion-induced iron overload, the Korean Society of Hematology Aplastic Anemia Working Party (KSHAAWP) reviewed the general consensus on iron overload and the Korean data on the clinical benefits of iron chelation therapy, and developed a Korean guideline for the treatment of iron overload.

Cost effectiveness of deferasirox compared to desferrioxamine in the treatment of iron overload in lower-risk, transfusion-dependent myelodysplastic syndrome patients

Objective:The study evaluated the cost effectiveness of deferasirox (Exjade) compared to non-proprietary desferrioxamine (DFO) for the control of transfusional iron overload in lower risk myelodysplastic syndromes (MDS) patients. A UK National Health Service perspective was adopted.Methods:Recent clinical evidence has demonstrated the efficacy and safety of deferasirox in transfusion-dependent MDS patients with elevated serum ferritin levels. An economic model was used to extrapolate the clinical benefits of iron chelation therapy (ICT) in a cohort of lower risk MDS patients. Costs for drug acquisition, drug administration and monitoring, and quality of life (utility) outcomes associated with mode of drug administration were derived from a variety of sources. The incremental cost per QALY gained for deferasirox was estimated. Costs and outcomes were discounted at 3.5% in line with UK standards.Results:The base-case cost effectiveness of deferasirox versus DFO was estimated to be £20,822 per QALY gained, the key driver being the additional quality of life benefits associated with a simpler mode of administration for deferasirox. A mean survival benefit for both forms of ICT of 4.5 years was estimated. The results were sensitive to drug dose, days of DFO administration, and patient weight.Conclusions:In the UK, a cost per QALY below £20,000–30,000 is considered cost effective. Hence, the results from this economic analysis suggest deferasirox is cost effective in lower risk, transfusion-dependent, MDS patients. Limitations with the analysis include a lack of comparative randomised controlled trial evidence, in particular to differentiate survival and clinical outcomes for deferasirox and DFO.