Estimative of costs of two therapeutic schemes for aplastic anemia among young individuals
DOI:
https://doi.org/10.33448/rsd-v11i15.37128Keywords:
Anemia, Aplastic; Economic Evaluation; Pancytopenia; Adolescent; Child.Abstract
Introduction: Aplastic anemia (AA) is the deficiency of the production of blood cells, with pancytopenia and hypocellular marrow as a result. Eltrombopag® (EPAG), a synthetic thrombopoietin mimic, showed improvement in bone marrow, CD34 and blood precursor cell counts. Today, it is indicated as first-line therapy, in addition to Immunosuppressive Therapy (IST) with cyclosporine and horse Antithymocyte Globulin (ATG). The aim of this study is to compare costs and results of the main current therapeutic schemes for AA among young individuals. Method: This is a study to estimate costs and results of a therapeutic scheme using EPAG associated with IST and the management of complications in comparison with standard therapy. The protocols used were those of the Ministry of Health and the scheme indicated in the literature for the EPAG. As a basis for calculation, a typical patient was simulated, who would be followed up by a pediatric hematology service in Sergipe, which works as part of the Unified Health System. Result: The cost of Cyclosporine + rabbit ATG treatment for 6 months, based on patient weight, was US$4500. The cost of Cyclosporine + rabbit GAT + EPAG treatment for 6 months was US$182,000. Conclusion: It is concluded, therefore, that drug therapy with EPAG increases the cost of treatment by 40 times. Several studies, however, point to clinical and laboratory gains and increased response rates to the proposed therapy, thus contributing to a better quality of life for a pediatric patient with AA.
References
Arcuri, L. J., Nabhan, S. K., Cunha, R., Nichele, S., Ribeiro, A. A. F., Fernandes, J. F., Daudt, L. E., Rodrigues, A. L. M., Arrais-Rodrigues, C., Seber, A., Atta, E. H., de Oliveira, J. S. R., Funke, V. A. M., Loth, G., Junior, L. G. D., Paz, A., Calixto, R. F., Gomes, A. A., Araujo, C. E. S., … Bonfim, C. (2020). Impact of CD34 Cell Dose and Conditioning Regimen on Outcomes after Haploidentical Donor Hematopoietic Stem Cell Transplantation with Post-Transplantation Cyclophosphamide for Relapsed/Refractory Severe Aplastic Anemia. Biology of Blood and Marrow Transplantation, 26(12), 2311–2317. https://doi.org/10.1016/j.bbmt.2020.09.007
Darrigo, L. G., Colturato, V., de Souza, M. P., Loth, G., Calixto, R., Seber, A., Zecchin, V. G., Esteves Daudt, L., Tavares, R. B., Arcuri, L., de Macedo, A. V., Vieira, A. K., Kuwahara, C., Ribeiro, L., Fernandes, J. F., Flowers, M. E., Pasquini, R., & Bonfim, C. (2019). Allogeneic Bone Marrow Transplants for Pediatric Severe Aplastic Anemia: Real-world Data comparing Matched Related and Unrelated Donors in a Developing Country. Retrospective study on behalf of the Pediatric Hematopoietic Stem Cell Transplant Working Grou. Pediatric Transplantation, 23(7), 1–7. https://doi.org/10.1111/petr.13552
Fang, M., Song, H., Zhang, J., Li, S., Shen, D., & Tang, Y. (2021). Efficacy and safety of immunosuppressive therapy with or without eltrombopag in pediatric patients with acquired aplastic anemia: A Chinese retrospective study. Pediatric Hematology and Oncology, 38(7), 633–646. https://doi.org/10.1080/08880018.2021.1895924
Groarke, E. M., Patel, B. A., Gutierrez-rodrigues, F., Rios, O., Lotter, J., Baldoni, D., Pierre, A. S., Shalhoub, R., Wu, C. O., Townsley, D. M., & Young, N. S. (2021). HHS Public Access. 192(3), 605–614. https://doi.org/10.1111/bjh.17232.Eltrombopag
Huan Ng, C., Jang-Milligan, F., & Schultz, K. R. (2021). Has Eltrombopag eliminated the need to use allogeneic HSCT in first line treatment of pediatric aplastic anemia? Pediatric Hematology and Oncology, 38(5), 417–419. https://doi.org/10.1080/08880018.2021.1940579
Iftikhar, R., Ahmad, P., de Latour, R., Dufour, C., Risitano, A., Chaudhri, N., Bazarbachi, A., De La Fuente, J., Höchsmann, B., Osman Ahmed, S., Gergis, U., Elhaddad, A., Halkes, C., Albeirouti, B., Alotaibi, S., Kulasekararaj, A., Alzahrani, H., Ben Othman, T., Cesaro, S., … de Latour, R. (2021). Special issues related to the diagnosis and management of acquired aplastic anemia in countries with restricted resources, a report on behalf of the Eastern Mediterranean blood and marrow transplantation (EMBMT) group and severe aplastic anemia working pa. Bone Marrow Transplantation, 56(10), 2518–2532. https://doi.org/10.1038/s41409-021-01332-8
Imada, K., Obara, N., Iida, H., Imajo, K., Maeda, T., Usuki, K., Fanghong, Z., Hombo, Y., Tajima, T., Kumagai, A., Matsuda, A., & Nakao, S. (2021). Eltrombopag in combination with rabbit anti-thymocyte globulin/cyclosporine a in immunosuppressive therapy-naïve patients with aplastic anemia in japan. Internal Medicine, 60(8), 1159–1168. https://doi.org/10.2169/internalmedicine.6063-20
Jie, M., Fu, L., Li, S., He, Y., Yao, J., Cheng, X., Zhang, L., Zheng, J., Zhang, R., & Wu, R. (2021). Efficacy and safety of eltrombopag in the first-line therapy of severe aplastic anemia in children. Pediatric Hematology and Oncology, 38(7), 647–657. https://doi.org/10.1080/08880018.2021.1900475
Kako, S., Kanda, Y., Onizuka, M., Aotsuka, N., Usuki, K., Tachibana, T., Kobayashi, T., Kato, J., Yano, S., Shimizu, H., Shono, K., Tanaka, M., Tsukamoto, S., Mori, T., Yamazaki, E., Najima, Y., Hangaishi, A., Hoshino, T., Watanabe, R., … Okamoto, S. (2020). Allogeneic hematopoietic stem cell transplantation for aplastic anemia with pre-transplant conditioning using fludarabine, reduced-dose cyclophosphamide, and low-dose thymoglobulin: A KSGCT prospective study. American Journal of Hematology, 95(3), 251–257. https://doi.org/10.1002/ajh.25693
Kuter, D. J. (2021). The structure, function, and clinical use of the thrombopoietin receptor agonist avatrombopag. Blood Reviews, xxxx, 100909. https://doi.org/10.1016/j.blre.2021.100909
Miguel, F. H., Zardetti, R. M., Carmo, S. S., Lima, V. da S., Marson, R. F., Machado, A. M., & Machado, A. R. da S. R. (2016). Aplasia de Medula Óssea: características, diagnóstico e tratamento. Revista Conexão Eletrônica, 13.
Moraz, G., Garcez, A. da S., de Assis, E. M., dos Santos, J. P., Barcellos, N. T., & Kroeff, L. R. (2015). Cost-effectiveness in health in Brazil: A systematic review. Ciencia e Saude Coletiva, 20(10), 3211–3229. https://doi.org/10.1590/1413-812320152010.00962015
Organization, W. H. (2007). Growth reference data. https://www.who.int/toolkits/growth-reference-data-for-5to19-years
Saúde, M. da. (2010). Portaria n° 940. https://bvsms.saude.gov.br/bvs/saudelegis/sas/2010/prt0490_23_09_2010.html
Scheinberg, P. (2011). Tratamento atual da anemia aplástica adquirida grave. 9(2), 229–235.
Scheinberg, P. (2021). Acquired severe aplastic anaemia: how medical therapy evolved in the 20th and 21st centuries. British Journal of Haematology, 194(6), 954–969. https://doi.org/10.1111/bjh.17403
Tremblay, G., Said, Q., Roy, A. N., Cai, B., Garib, S. A., Hearnden, J., & Forsythe, A. (2019). Budget impact of eltrombopag as first-line treatment for severe aplastic anemia in the United States. ClinicoEconomics and Outcomes Research, 11, 673–681. https://doi.org/10.2147/CEOR.S226323
Usuki, K. (2016). Aplastic anemia. [Rinsho Ketsueki] The Japanese Journal of Clinical Hematology, 57(10), 1890–1899. https://doi.org/10.1182/blood.v3.2.192.192
Verghese. (2011). 基因的改变NIH Public Access. Bone, 23(1), 1–7. https://doi.org/10.1016/j.pcl.2013.08.011.Acquired
Zhang, M. X., Wang, Q., & Wang, X. Q. (2021). Hematopoietic stem-cell transplantation versus immunosuppressive therapy in patients with adult acquired severe aplastic anemia: A cost-effectiveness analysis. International Journal of General Medicine, 14, 3529–3537. https://doi.org/10.2147/IJGM.S310844
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