Economic and clinical burden of managing transfusion-dependent β-thalassemia in the United States

References

• Modell B, Darlison M. Global epidemiology of haemoglobin disorders and derived service indicators. Bull World Health Organ. 2008;86(6):480–487. doi: 10.2471/blt.06.036673.
   PubMed Web of Science ®Google Scholar
• Taher AT, Musallam KM, Cappellini MD. Beta-thalassemias. N Engl J Med. 2021;384(8):727–743. doi: 10.1056/NEJMra2021838.
   PubMed Web of Science ®Google Scholar
• Galanello R, Origa R. Beta-thalassemia. Orphanet J Rare Dis. 2010;5:11. doi: 10.1186/1750-1172-5-11.
   PubMed Web of Science ®Google Scholar
• Kattamis A, Forni GL, Aydinok Y, et al. Changing patterns in the epidemiology of beta-thalassemia. Eur J Haematol. 2020;105(6):692–703. doi: 10.1111/ejh.13512.
   PubMed Web of Science ®Google Scholar
• Sayani FA, Kwiatkowski JL. Increasing prevalence of thalassemia in America: implications for primary care. Ann Med. 2015;47(7):592–604. doi: 10.3109/07853890.2015.1091942.
   PubMed Web of Science ®Google Scholar
• Lal A, Wong T, Keel S, et al. The transfusion management of beta thalassemia in the United States. Transfusion. 2021;61(10):3027–3039. doi: 10.1111/trf.16640.
   PubMed Web of Science ®Google Scholar
• Lal A, Wong TE, Andrews J, et al. Transfusion practices and complications in thalassemia. Transfusion. 2018;58(12):2826–2835. doi: 10.1111/trf.14875.
   PubMed Web of Science ®Google Scholar
• Taher AT, Weatherall DJ, Cappellini MD. Thalassaemia. Lancet. 2018;391(10116):155–167. doi: 10.1016/S0140-6736(17)31822-6.
   PubMed Web of Science ®Google Scholar
• Cappellini MD, Farmakis D, Porter J, et al. 2021 Guidelines for the management of transfusion dependent thalassaemia (TDT). Nicosia (Cyprus): Thalassaemia International Federation; 2021.
   Google Scholar
• Tang CH, Furnback W, Wang BCM, et al. Relationship between transfusion burden, healthcare resource utilization, and complications in patients with beta-thalassemia in Taiwan: a real-world analysis. Transfusion. 2021;61(10):2906–2917. doi: 10.1111/trf.16636.
   PubMed Web of Science ®Google Scholar
• Alshamsi S, Hamidi S, Narci HO. Healthcare resource utilization and direct costs of transfusion-dependent thalassemia patients in Ddubai, United Arab Emirates: a retrospective cost-of-illness study. BMC Health Serv Res. 2022;22(1):304. doi: 10.1186/s12913-022-07663-6.
   PubMed Web of Science ®Google Scholar
• Weiss M, Parisi Jun M, Sheth S. Clinical and economic burden of regularly transfused adult patients with beta-thalassemia in the United States: a retrospective cohort study using payer claims. Am J Hematol. 2019;94(5):E129–E132. doi: 10.1002/ajh.25429.
   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(4):263–270. doi: 10.1002/ajh.21049.
   PubMed Web of Science ®Google Scholar
• Udeze C, Evans KA, Yang Y, et al. Economic and clinical burden of managing sickle cell disease with recurrent vaso-occlusive crises in the United States. Adv Ther. 2023;40(8):3543–3558. doi: 10.1007/s12325-023-02545-7.
   PubMed Web of Science ®Google Scholar
• Tan L, Reibman J, Ambrose C, et al. Clinical and economic burden of uncontrolled severe noneosinophilic asthma. Am J Manag Care. 2022;28(6):e212–e220. doi: 10.37765/ajmc.2022.89159.
   PubMed Web of Science ®Google Scholar
• Zeiger R, Sullivan P, Chung Y, et al. Systemic corticosteroid-related complications and costs in adults with persistent asthma. J Allergy Clin Immunol Pract. 2020;8(10):3455–3465.e13. doi: 10.1016/j.jaip.2020.06.055.
   PubMed Web of Science ®Google Scholar
• BLS.gov [Internet]. Washington (DC): U.S. Bureau of Labor Statistics; c2023 [cited 2023 Mar 14]. Available from: https://www.bls.gov/cpi/factsheets/medical-care.htm.
   Google Scholar
• Vichinsky E, El-Beshlawy A, Al Zoebie A, et al. Long-term safety and efficacy of deferasirox in young pediatric patients with transfusional hemosiderosis: results from a 5-year observational study (ENTRUST). Pediatr Blood Cancer. 2017;64(9):8. doi: 10.1002/pbc.26507.
   Web of Science ®Google Scholar
• Udeze C, Maruszczyk K, Atter M, et al. Projected lifetime economic burden of transfusion dependent beta-thalassemia in the United States. HemaSphere. 2022;6:2208–2209. doi: 10.1097/01.HS9.0000852180.45597.87.
   Google Scholar
• Johnson KM, Jiao B, Ramsey SD, et al. Lifetime medical costs attributable to sickle cell disease among nonelderly individuals with commercial insurance. Blood Adv. 2023;7(3):365–374. doi: 10.1182/bloodadvances.2021006281.
   PubMed Web of Science ®Google Scholar
• Locatelli F, Thompson AA, Kwiatkowski JL, et al. Betibeglogene autotemcel gene therapy for non-β(0)/β(0) genotype β-thalassemia. N Engl J Med. 2022;386(5):415–427. doi: 10.1056/NEJMoa2113206.
   PubMed Web of Science ®Google Scholar
• Manjelievskaia J, Boytsov N, Brouillette MA, et al. The direct and indirect costs of adult atopic dermatitis. J Manag Care Spec Pharm. 2021;27(10):1416–1425. doi: 10.18553/jmcp.2021.27.10.1416.
   PubMed Web of Science ®Google Scholar
• Varnado OJ, Manjelievskaia J, Wenyu Y, et al. Health care resource utilization and costs associated with treatment among patients initiating calcitonin gene-related peptide inhibitors vs other preventive migraine treatments in the United States. J Manag Care Spec Pharm. 2022;28(8):818–829. doi: 10.18553/jmcp.2022.28.8.818.
   PubMed Web of Science ®Google Scholar
• Fenske DC, Ding Y, Morrow P, et al. Comparing the burden of illness in patients with alopecia areata vs atopic dermatitis in the US population from a payer perspective. J Manag Care Spec Pharm. 2023;29(4):409–419. doi: 10.18553/jmcp.2023.29.4.409.
   PubMed Web of Science ®Google Scholar
• AHRQ.gov [Internet]. Rockville (MD): Agency for Healthcare Research and Quality; c2023 [cited 2023 May 19]. Available from: https://meps.ahrq.gov/about_meps/Price_Index.shtml.
   Google Scholar