Comprehensive economic studies on sickle cell anemia treatments

Integrative literature review developed by collecting data in the electronic databases National Library of Medicine - Medline via PubMed; Elsevier's Scopus; Current Index to Nursing and Allied Health Literature; Science Direct and Web of Science with descriptors indexed in Medical Subject Headings. The studies were selected by the relevance test and analyzed according to the classification of economic analyses in health and the classification system of evidence quality and strength of recommendations.

Results:

Nine articles were part of this review, seven of which were retrieved from Elsevier's Scopus and two from Medline via PubMed. All articles reported on studies focusing on the perspectives of using hydroxyurea and blood transfusion in the treatment of sickle cell anemia.

Conclusion:

No studies were identified that were conducted in Brazil and involved this type of analysis for sickle cell anemia. Much remains to be done worldwide to assess existing technologies, reassess the technologies currently used and implement continuous diagnosis and treatment, by means of a system that guarantees an active and efficient care network for the patients.

Keywords
Sickle cell disease; Hemoglobin, sickle; Health economics; Costs and cost analysis; Cost-benefit analysis

Author/year	Type of economic analysis	Classification GRADE	Methodological design	Recommendations and limitations
Moore et al., 2000⁽¹⁶16. Moore RD, Charache S, Terrin ML, Barton FB, Ballas SK. Cost-effectiveness of hydroxyurea in sickle cell anemia. Investigators of the Multicenter Study of Hydroxyurea in Sickle Cell Anemia. Am J Hematol. 2000;64(1):26-31.⁾	Cost-effectiveness	Moderate	Based on a randomized controlled clinical trial in pediatrics (N = 299). Wilcoxon test. Control group (N = 147) and HU Group (N = 152)	HU decreased painful seizures in sickle cell anemia (SCA) and reduced hospitalization costs, a fact that compensated for the use, but requires clinical monitoring and investigations. Limitations: quality of life or years of life saved were not evaluated.
Stallwort; Jerrell; Tripathi, 2010⁽¹⁷17. Stallworth JR, Jerrell JM, Tripath IV. Cost-effectiveness of hydroxyurea in reducing the frequency of pain episodes and hospitalization in pediatric sickle cell disease. Am J Hematol. 2010;85(10):795-97.⁾	Cost-effectiveness	Moderate	Based on pediatric retrospective cohort (N = 523). Rate ratio. Control group (N = 348) and HU Group (N = 175)	Need for new long-term observational research for the use of HU in children. Lesser pain episodes, hospitalizations and total costs. Limitations: retrospective data, not controlled, without interviews and analysis of dropouts.
Prados et al., 2010⁽¹⁸18. Prados MBV, De Jesus RG, Sein AF, Garcia MI. Efficacy, safety and cost-efficiency of using na alternative technique for automated exchange transfusion in pediatric patients with sickle cell disease. Bol Asoc Med P R. 2010;102(1):13-7.⁾	Cost-minimization	Low	Based on pediatric retrospective cohort (N = 14). Non-paired t test. Pump method (N = 10) group and automated blood cell separator (N = 4) group	Exchange transfusion using a pump is realistic, economical and effective. Ideal for social contexts with difficulties. Limitations: retrospective data from a hospital and small sample.
Cherry et al., 2012⁽¹⁹19. Cherry MG, Greenhalgh J, Osipenko L, Venkatachalam M, Boland A, Dundar Y, et al. The clinical effectiveness and cost-effectiveness of primary stroke prevention in children with sickle cell disease: a systematic review and economic evaluation. Health Technol Assess. 2012;16(43)1-125.⁾	Cost-utility	Moderate	Pediatric hypothetical cohort (N = 1000). Markov model. Quality Adjusted Life Years (QALYs). Transcranial Doppler (DTC) altered with transfusion group and DTC altered without transfusion group.	The practice of DTC and identification of the risk for stroke with blood transfusion seems to be cost-effective, improving the living and development condition. Limitations: few clinical data related to blood transfusion in stroke prevention.
Spackman et al., 2014⁽²⁰20. Spackman E, Sculpher H, Howard J, Malfroy H, Llewelyn C, Choo G, et al. Cost-effectiveness analysis of preoperative transfusion in patients with sickle cell disease using evidence from the TAPS trial. Eur J Haematol. 2014;92(3):249-55.⁾	Cost-utility	Moderate	Based on a randomized, adult and pediatric study (N = 70). Monte Carlo simulation. QALYs Preoperative blood transfusion group and control group.	Preoperative blood transfusion seems to be cost-effective in low-cost and moderate-risk surgeries. Increased acute chest syndrome in control group with direct effects in the short term. Limitations: number of patients, lack of QALYs in children.
Kacker et al., 2014⁽²¹21. Kacker S, Ness PM, Savage WJ, Frick KD, Shirey RS, King KE, et al. Economic evaluation of a hypothetical screening assay for alloimmunization risk among transfused patients with sickle cell disease. Transfusion. 2014;54(8):2034-44.⁾	Cost-effectiveness	Moderate	Hypothetical cohort (N = 8000). Markov model. Prospective antigen matching group, history-based antigen group, perfect matching group and imperfect antigen matching group.	A specific screening test for alloimmunization, even if imperfect could provide support for clinical benefits in blood transfusion. Limitations: simplified alloimmunization model, did not incorporate dynamic population and represents local realities.
Kacker et al., 2014⁽²²22. Kacker S, Ness PM, Savage WJ, Frick KD, Shirey RS, King KE, et al. Cost-effectiveness of prospective red blood cell antigen matching to prevent alloimmunization among sickle cell patients. Transfusion. 2014;54(1):86-97.⁾	Cost-effectiveness	Moderate	Hypothetical cohort (N = 8500). Markov model. Antigen group based on history and prospective antigen matching group.	The cost of specific antigen tests for alloimmunization would be high. Need for research in the area. Limitations: simple model, underestimated costs, no indirect costs.
Cunningham-Myrie et al., 2015⁽²³23. Cunningham-Myrie C, Abdulkadri A, Waugh A, Bortolusso AS, King LG, Knight-Madden J, et al. Hydroxyurea use in prevention of stroke recurrence in children with sickle cell disease in a developing country: A cost effectiveness analysis. Pediatr Blood Cancer. 2015;62(10):1862-4.⁾	Cost-effectiveness	Moderate	Based on a pediatric cohort study (N = 42). Incremental cost-effectiveness and student's t test. Control group (N = 32) and hydroxyurea group (N = 10).	HU is cost effective for preventing recurrent strokes. Can be beneficial in environments with limited resources. Limitations: analysis restricted to direct costs, without indirect costs.
Kuznik et al., 2016⁽²⁴24. Kuznik A, Habibi AG, Munube D, Lamorde M. Newborn screening and prophylactic interventions for sickle cell disease in 47 countries in sub-Saharan Africa: a cost-effectiveness analysis. BMC Health Serv Res. 2016;16(304):1-12.⁾	Cost-effectiveness	Moderate	Pediatric hypothetical cohort (N = 228,169). Markov model and disability-adjusted life years (DALYs). Control group and neonatal screening group and prophylactic intervention.	Neonatal screening and prophylactic prevention would be cost-effective in some countries with higher incidence rates of the disease. Specialized diagnosis and treatment centers are needed. Limitations: non-generalizable estimates and costs. Did not observe the intervention throughout life.

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[1] Corresponding author Andréia Insabralde de Queiroz Cardoso, E-mail: andreia.cardoso@ufms.br