Iron chelation therapy: Clinical effectiveness, economic burden and quality of life in patients with iron overload

Payne, Krista A.; Rofail, Diana; Baladi, Jean-Francois; Viala, Muriel; Abetz, Linda; Desrosiers, Marie-Pierre; Lordan, N; Ishak, K. Jack; Proskorovsky, Irina

doi:10.1007/s12325-008-0085-z

Cited by 57 publications

(46 citation statements)

References 37 publications

(34 reference statements)

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“…8 Deferoxamine (DFO) is an effective iron chelator, but its demanding treatment regimen can result in suboptimal compliance, with ultimate failure to show clinically meaningful decreases in serum ferritin levels, as well as impaired quality of life. 9 A few studies have been conducted with DFO in patients with AA; however, these studies only enrolled a small number of patients (n ϭ 1-33); therefore, data with DFO in this population remain limited. 5,10,11 Deferasirox is a once-daily, oral, iron chelator approved for the treatment of chronic iron overload resulting from blood transfusions.…”

Section: Introductionmentioning

confidence: 99%

Iron chelation therapy with deferasirox in patients with aplastic anemia: a subgroup analysis of 116 patients from the EPIC trial

Lee

Yoon²,

Shen

et al. 2010

Blood

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The prospective 1-year Evaluation of Patients' Iron Chelation with Exjade (EPIC) study enrolled a large cohort of 116 patients with aplastic anemia; the present analyses evaluated the efficacy and safety of deferasirox in this patient population. After 1 year, median serum ferritin decreased significantly from 3254 ng/mL at baseline to 1854 ng/mL (P < .001). Decreases occurred in chelation-naive (3229-1520 ng/mL; P < .001, last-observation-carried-forward analysis), and previously chelated (3263-2585 ng/mL; P ‫؍‬ .21, last-observationcarried-forward analysis) patients and were reflective of dose adjustments and ongoing iron intake. Baseline labile plasma iron levels were within normal range despite high serum ferritin levels. The most common drug-related adverse events were nausea (22%) and diarrhea (16%). Serum creatinine increases more than 33% above baseline and the upper limit of normal occurred in 29 patients (25%), but there were no progressive increases; concomitant use of cyclosporine had a significant impact on serum creatinine levels. The decrease in mean alanine aminotransferase levels at 1 year correlated significantly with reduction in serum ferritin (r ‫؍‬ 0.40, P < .001). Mean absolute neutrophil and platelet counts remained stable during treatment, and there were no drug-related cytopenias. This prospective dataset confirms the efficacy and well characterizes the tolerability profile of deferasirox in a large population of patients with aplastic anemia. This study was registered at www.clinicaltrials. gov as #NCT00171821. (Blood. 2010;116(14):2448-2454)

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Section: Introductionmentioning

confidence: 99%

Iron chelation therapy with deferasirox in patients with aplastic anemia: a subgroup analysis of 116 patients from the EPIC trial

Lee

Yoon²,

Shen

et al. 2010

Blood

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“…Another UK-based study transformed SF-36 utility scores to utilities among patients treated with ICT (DFO, deferiprone or a combination of DFO and deferiprone) and reported an average utility of 0.66 (range: 0.37-0.96) associated with the use of ICT. [38] As observed in the McLeod et al [35] study, this variation in utility values and methods can significantly affect the calculated ICERs. As a result, careful documentation of the source of utility values, rationale for selecting a particular utility estimate and sensitivity analyses to evaluate the impact of change in utility estimates is important in any cost-effectiveness assessment of ICTs.…”

Section: Discussionmentioning

confidence: 91%

Pharmacoeconomic Considerations in Treating Iron Overload in Patients with β-Thalassaemia, Sickle Cell Disease and Myelodysplastic Syndromes in the US

Zhang

Donga

Corral³

et al. 2011

PharmacoEconomics

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Patients with β-thalassaemia, sickle cell disease (SCD) and myelodysplastic syndromes (MDS) require chronic blood transfusions, which can lead to iron overload and substantial morbidity and mortality. To reduce the excess iron and its deleterious effects, available iron chelation therapy (ICT) in the US includes oral deferasirox or infusional deferoxamine (DFO). The aim of this study was to review and synthesize the available pharmacoeconomic evidence on ICT in patients with β-thalassaemia, SCD and MDS in the US. We systematically identified and reviewed pharmacoeconomic studies of ICT in patients with β-thalassaemia, SCD and MDS that either were published in MEDLINE-indexed, English-language journals from 1999 to 2009, or appeared in medical society websites and scientific meeting abstracts. We assessed available cost-of-illness, cost-of-treatment, cost-consequence, cost-effectiveness, utility and patient-satisfaction studies. The majority of the 20 identified studies assessed cost of treatment, mainly focusing on acquisition and administration costs of ICTs. Gaps in the published literature include current data on direct medical costs for patients with MDS, direct medical costs associated with complications of iron overload, direct non-medical costs, indirect costs and patient utilities. Different underlying model assumptions, methodologies and comparators were found in the cost-effectiveness studies, which yielded a broad range of incremental cost-effectiveness ratios for different ICTs. Comprehensive cost-of-illness studies are needed to address data gaps in the published literature regarding the economic burden of iron overload. Comparative-effectiveness studies that evaluate clinical, economic and patient-reported outcomes would help the medical community to better understand the value of different ICTs.

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“…The economic, clinical, and humanistic impact of iron overload may become substantial, and poor adherence to ICTs has been shown to result in serum ferritin targets to not be achieved, which negatively impacts costs, morbitidy and mortality, and healthrelated quality of life [31,[40][41][42]. The long-term ramifications of nonadherence to ICTs in persons with iron overload may manifest in severe clinical complications, particularly hepatic failure, iron-induced cardiomyopathy, or pancreatic iron deposition [9].…”

Section: Cost Analysismentioning

confidence: 99%

Long-term persistency and costs associated with the use of iron chelation therapies in the treatment of Sickle cell disease within Medicaid programs

Armstrong

Skrepnek

Sasané

et al. 2012

Journal of Medical Economics

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Iron chelation therapy: Clinical effectiveness, economic burden and quality of life in patients with iron overload

Cited by 57 publications

References 37 publications

Iron chelation therapy with deferasirox in patients with aplastic anemia: a subgroup analysis of 116 patients from the EPIC trial

Iron chelation therapy with deferasirox in patients with aplastic anemia: a subgroup analysis of 116 patients from the EPIC trial

Pharmacoeconomic Considerations in Treating Iron Overload in Patients with β-Thalassaemia, Sickle Cell Disease and Myelodysplastic Syndromes in the US

Long-term persistency and costs associated with the use of iron chelation therapies in the treatment of Sickle cell disease within Medicaid programs

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