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

References

• Saunthararajah Y, Vichinsky EP. Sickle cell disease - clinical features and management. In: Hoffmann R, Benz EJ, Shattil SS, eds. Hematology: basic principles and practice. 5th ed. Philadelphia, PA: Elsevier Churchill Livingstone, 2008. p 577–601
   Google Scholar
• Roseff SD. Sickle cell disease: a review. Immunohematology 2009;25:67–74
   PubMedGoogle Scholar
• Brousseau DC, Panepinto JA, Nimmer M, et al. The number of people with sickle-cell disease in the United States: national and state estimates. Am J Hematol 2010;85:77–8
   PubMed Web of Science ®Google Scholar
• Ballas SK. The cost of health care for patients with sickle cell disease. Am J Hematol 2009;84:320–2
   PubMed Web of Science ®Google Scholar
• Redding-Lallinger R, Knoll C. Sickle cell disease–pathophysiology and treatment. Curr Probl Pediatr Adolesc Health Care 2006;36:346–76
   PubMedGoogle Scholar
• Strouse JJ, Jordan LC, Lanzkron S, et al. The excess burden of stroke in hospitalized adults with sickle cell disease. Am J Hematol 2009;84:548–52
   PubMed Web of Science ®Google Scholar
• Institute NHLaB. The management of sickle cell disease. 2002. http://www.nhlbi.nih.gov/health/prof/blood/sickle/sc_mngt.pdf, accessed April 4, 2012
   Google Scholar
• Society SC. Standards for the clinical care of adjust with sickle cell disease in the UK. 2008. http://www.sicklecellsociety.org/app/webroot/files/files/CareBook.pdf, accessed April 4, 2012
   Google Scholar
• Brittenham GM. Iron-chelating therapy for transfusional iron overload. N Engl J Med 2011;364:146–56
   PubMed Web of Science ®Google Scholar
• Fung EB, Harmatz P, Milet M, et al. Morbidity and mortality in chronically transfused subjects with thalassemia and sickle cell disease: a report from the multi-center study of iron overload. Am J Hematol 2007;82:255–65
   PubMed Web of Science ®Google Scholar
• Ballas SK. Iron overload is a determinant of morbidity and mortality in adult patients with sickle cell disease. Semin Hematol 2001;38(1 Suppl):30–6
   PubMedGoogle Scholar
• Yang LP, Keam SJ, Keating GM. Deferasirox: a review of its use in the management of transfusional chronic iron overload. Drugs 2007;67:2211–30
   PubMed Web of Science ®Google Scholar
• Cappellini MD, Bejaoui M, Agaoglu L, et al. Prospective evaluation of patient-reported outcomes during treatment with deferasirox or deferoxamine for iron overload in patients with beta-thalassemia. Clin Ther 2007;29:909–17
   PubMed Web of Science ®Google Scholar
• Vichinsky E, Pakbaz Z, Onyekwere O, et al. Patient-reported outcomes of deferasirox (Exjade, ICL670) versus deferoxamine in sickle cell disease patients with transfusional hemosiderosis. Substudy of a randomized open-label phase II trial. Acta Haematol 2008;119:133–41
   PubMed Web of Science ®Google Scholar
• Jordan LB, Vekeman F, Sengupta A, et al. Persistence and compliance of deferoxamine versus deferasirox in Medicaid patients with sickle-cell disease. J Clin Pharm Ther 2012;37:173–81
   PubMed Web of Science ®Google Scholar
• Betito E. ApoPharma announces FDA advisory committee recommendation in favor of Ferriprox® (deferiprone) approval. 2011. http://www.apotex.com/global/about/press/20110914.asp, accessed April 4, 2012
   Google Scholar
• Zhang B, Donga PZ, Corral M, et al. Pharmacoeconomic considerations in treating iron overload in patients with beta-thalassaemia, sickle cell disease and myelodysplastic syndromes in the US: a literature review. Pharmacoeconomics 2011;29:461–74
   PubMedGoogle Scholar
• Imran F, Phatak P. Pharmacoeconomic benefits of deferasirox in the management of iron overload syndromes. Expert Rev Pharmacoecon Outcomes Res 2009;9:297–304
   PubMedGoogle Scholar
• Delea TE, Edelsberg J, Sofrygin O, et al. Consequences and costs of noncompliance with iron chelation therapy in patients with transfusion-dependent thalassemia: a literature review. Transfusion 2007;47:1919–29
   PubMed Web of Science ®Google Scholar
• Delea TE, Hagiwara M, Thomas SK, et al. Outcomes, utilization, and costs among thalassemia and sickle cell disease patients receiving deferoxamine therapy in the United States. Am J Hematol 2008;83:263–70
   PubMed Web of Science ®Google Scholar
• Delea TE, Sofrygin O, Thomas SK, et al. Cost effectiveness of once-daily oral chelation therapy with deferasirox versus infusional deferoxamine in transfusion-dependent thalassaemia patients: US healthcare system perspective. Pharmacoeconomics 2007;25:329–42
   PubMed Web of Science ®Google Scholar
• Kamon J, Akehurst RL, Jewitt K, et al. Cost utility analysis of deferasirox versus deferoxamine (Desferal) for patients requiring iron chelation therapy in the United Kingdom. Haematologica 2007;92(1 Suppl):222–3
   PubMedGoogle Scholar
• Reuters T. MarketScan research databases. 2012. http://thomsonreuters.com/products_services/healthcare/healthcare_products/a-z/marketscan_research_analytics, accessed July 2, 2012
   Google Scholar
• Software A. 2012 ICD-9-CM Diagnosis Code 282.6. 2012. http://www.icd9data.com/2012/Volume1/280-289/282/282.6.htm, accessed July 2, 2012
   Google Scholar
• Steiner JF, Prochazka AV. The assessment of refill compliance using pharmacy records: methods, validity, and applications. J Clin Epidemiol 1997;50:105–16
   PubMed Web of Science ®Google Scholar
• Services CfMM. Healthcare Common Procedure Coding System (HCPCS). 2012. https://www.cms.gov/Medicare/Coding/MedHCPCSGenInfo/index.html?redirect=/MedHCPCSGenInfo/, accessed July 2, 2012
   Google Scholar
• Charlson ME, Pompei P, Ales KL, et al. A new method of classifying prognostic comorbidity in longitudinal studies: development and validation. J Chronic Dis 1987;40:373–83
   PubMedGoogle Scholar
• Deyo RA, Cherkin DC, Ciol MA. Adapting a clinical comorbidity index for use with ICD-9-CM administrative databases. J Clin Epidemiol 1992;45:613–9
   PubMed Web of Science ®Google Scholar
• Skrepnek GH, Olvey EL, Sahai A. Econometric approaches in evaluating costs and outcomes within pharmacoeconomic analyses. Pharm Policy Law 2012;14:105–22
   Google Scholar
• Hertz-Picciotto I, Rockhill B. Validity and efficiency of approximation methods for tied survival times in Cox regression. Biometrics 1997;53:1151–6
   PubMed Web of Science ®Google Scholar
• Skrepnek GH. Regression methods in the empiric analysis of health care data. J Manag Care Pharm 2005;11:240–51
   PubMedGoogle Scholar
• Grosse SD, Boulet SL, Amendah DD, et al. Administrative data sets and health services research on hemoglobinopathies: a review of the literature. Am J Prev Med 2010;38(4 Suppl):S557–S67
   Google Scholar
• Gabutti V, Piga A. Results of long-term iron-chelating therapy. Acta Haematol 1996;95:26–36.
   PubMed Web of Science ®Google Scholar
• Weissman L, Treadwell M, Vichinsky E. Evaluation of home desferal care in transfusion-dependent children with thalassemia and sickle cell disease (SCD). J Pediatr Hematol Oncol 1996;18:454–5
   Google Scholar
• Trachtenberg F, Vichinsky E, Haines D, et al. Iron chelation adherence to deferoxamine and deferasirox in thalassemia. Am J Hematol 2011;86:433–6
   PubMed Web of Science ®Google Scholar
• Alvarez O, Rodriguez-Cortes H, Robinson N, et al. Adherence to deferasirox in children and adolescents with sickle cell disease during 1-year of therapy. J Pediatr Hematol Oncol 2009;31:739–44
   PubMedGoogle Scholar
• Raphael JL, Bernhardt MB, Mahoney DH, et al. Oral iron chelation and the treatment of iron overload in a pediatric hematology center. Pediatr Blood Cancer 2009;52:616–20
   PubMed Web of Science ®Google Scholar
• Nietert PJ, Silverstein MD, Abboud MR. Sickle cell anaemia: epidemiology and cost of illness. Pharmacoeconomics 2002;20:357–66
   PubMed Web of Science ®Google Scholar
• Kauf TL, Coates TD, Huazhi L, et al. The cost of health care for children and adults with sickle cell disease. Am J Hematol 2009;84:323–7
   PubMed Web of Science ®Google Scholar
• Lanzkron S, Haywood C, Jr Segal JB, et al. Hospitalization rates and costs of care of patients with sickle-cell anemia in the state of Maryland in the era of hydroxyurea. Am J Hematol 2006;81:927–32
   PubMed Web of Science ®Google Scholar
• Mvundura M, Amendah D, Kavanagh PL, et al. Health care utilization and expenditures for privately and publicly insured children with sickle cell disease in the United States. Pediatr Blood Cancer 2009;53:642–6
   PubMed Web of Science ®Google Scholar
• Amendah DD, Mvundura M, Kavanagh PL, et al. Sickle cell disease-related pediatric medical expenditures in the U.S. Am J Prev Med 2010;38(4 Suppl):S550–6
   PubMed Web of Science ®Google Scholar
• Skrepnek GH. Cost-effectiveness analysis. In: Bootman JL, Townsend RJ, McGhan WF, eds. Principles of Pharmacoeconomics. 3rd ed. Cincinnati, OH: Harvey Whitney Books Company, 2005. p 82–116
   Google Scholar
• Yusuf HR, Lloyd-Puryear MA, Grant AM, et al. Sickle cell disease: the need for a public health agenda. Am J Prev Med 2011;41(6 Suppl 4):S376–83
   PubMedGoogle Scholar
• Hulihan MM, Sayers CA, Grosse SD, et al. Iron overload: what is the role of public health? Am J Prev Med 2011;41(6 Suppl 4):S422–7
   Google Scholar
• Payne KA, Rofail D, Baladi JF, et al. Iron chelation therapy: clinical effectiveness, economic burden and quality of life in patients with iron overload. Adv Ther 2008;25:725–42
   PubMed Web of Science ®Google Scholar
• Abetz L, Baladi JF, Jones P, et al. The impact of iron overload and its treatment on quality of life: results from a literature review. Health Qual Life Outcomes 2006;4:73
   PubMed Web of Science ®Google Scholar
• Porter JB, Evangeli M, El-Beshlawy A. Challenges of adherence and persistence with iron chelation therapy. Int J Hematol 2011;94:453–60
   PubMed Web of Science ®Google Scholar
• Boulet SL, Yanni EA, Creary MS, et al. Health status and healthcare use in a national sample of children with sickle cell disease. Am J Prev Med 2010;38(4 Suppl):S528–35
   Google Scholar