Anemia aplásica: un nuevo reto farmacológico en la práctica clínica
| Issue | Vol. 7 Núm. 1 (2023): Ciencia y Salud, enero-marzo |
| DOI | |
| Publicado | mar 9, 2023 |
|
Estadísticas |
Universidad Pontificia Bolivariana, Medellín, Colombia
Resumen
Introducción: la anemia aplásica (AA) es una enfermedad poco frecuente, caracterizada por presentar una insuficiencia en la médula ósea y una pancitopenia, sin rastro de procesos mieloproliferativos o fibróticos.
Objetivo: conocer los diversos tratamientos farmacológicos usados en la terapia inmunosupresora (IST) en la AA; adicionalmente, se profundizará en la respuesta de los pacientes frente a la IST y los mecanismos de acción de los fármacos utilizados.
Metodología: se realizó una búsqueda sistemática en las bases de datos de literatura médica como PUBMED, British Medical Journal, New England Journal, entre otros. Se incluyeron artículos tanto en inglés como en español.
Conclusiones: el manejo de la anemia aplásica continúa representando un reto para la medicina moderna; no obstante, se ha desarrollado un gran número de nuevas opciones terapéuticas para tratar a los pacientes.
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Akram Z, Ahmed P, Kajigaya S, Satti TM, Satti HS, Chaudhary QUN, et al. Epidemiological, clinical and genetic characterization of aplastic anemia patients in Pakistan. Ann Hematol. 2019;98(2):301-12. doi: 10.1007/s00277-018-3542-z
Ally M, Magesa P, Luzzatto L. High frequency of acquired aplastic anemia in Tanzania. Am J Hematol. 2019;94(4):E86-E88. doi: 10.1002/ajh. 25388
John CO, Korubo K, Ogu R, Mmom CF, Mba AG, Chidiadi EA, et al. Management of aplastic anaemia in pregnancy in a resource poor centre. The Pan African Medical Journal. 2016;24:277. doi: 10.11604/pamj.2016.24.277.9880
Ahmed P, Chaudhry QUN, Satti TM, Mahmood SK, Ghafoor T, Shahbaz N, et al. Epidemiology of aplastic anemia: a study of 1324 cases. Hematology. 2020;25(1):48-54. doi: 10.1080/ 16078454.2019.1711344
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Dong X, Han Y, Abeysekera IR, Shao Z, Wang H. GDF11 is increased in patients with aplastic anemia. Hematology. 2019;24(1):331-6. doi: 10.10 80/16078454.2019.1574386
Furlong E, Carter T. Aplastic anaemia: Current concepts in diagnosis and management. J Paediatr Child Health. 2020;56(7):1023-8. doi: 10.1111/jpc. 14996
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Ebens CL, DeFor TE, Tryon R, Wagner JE, MacMillan ML. Comparable Outcomes after HLA-Matched Sibling and Alternative Donor Hematopoietic Cell Transplantation for Children with Fanconi Anemia and Severe Aplastic Anemia. Biol Blood Marrow Transplant.2018;24(4):765-71. doi: 10.1016/j.bbmt.2017.11.031
Cooper JP, Farah RJ, Stevenson PA, Gooley TA, Storb R, Scott BL. Hematopoietic Cell Transplantation for Paroxysmal Nocturnal Hemoglobinuria in the Age of Eculizumab. Biol Blood Marrow Transplant. 2019;25(7):1331-9. doi: 10.1016/j.bbmt. 2019.01.033
Li Y, Ding S, Liu C, Chen T, Liu H, Li L, et al. Abnormalities of quantities and functions of CD56bright natural killer cells in non-severe aplastic Anemia. Hematology. 2019;24(1):405-12. doi: 10.1080/16078454.2019.1590963
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Camitta BM, Rappeport JM, Parkman R, Nathan DG. Selection of patients for bone marrow transplantation in severe aplastic anemia. Blood. 1975;45(3):355-63.
Killick SB, Bown N, Cavenagh J, Dokal I, Foukaneli T, Hill A, et al. Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol. 2016;172(2):187-207. doi: 10.1111/bjh.13853
Lian Y, Shi J, Nie N, Huang Z, Shao Y, Zhang J, et al. Evolution patterns of paroxysmal nocturnal hemoglobinuria clone and clinical implications in acquired bone marrow failure. Exp Hematol. 2019;77:41-50. doi: 10.1016/j.exphem.2019.08.005
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Dufour C, Veys P, Carraro E, Bhatnagar N, Pillon M, Wynn R, et al. Similar outcome of upfront-unrelated and matched sibling stem cell transplantation in idiopathic paediatric aplastic anaemia. A study on behalf of the UK Paediatric BMT Working Party, Paediatric Diseases Working Party and Severe Aplastic Anaemia Working Party of EBMT. Br J Haematol. 2015;171(4):585-94. doi: 10.1111/bjh.13614
Rogers ZR, Nakano TA, Olson TS, Bertuch AA, Wang W, Gillio A, et al. Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium s10.3324/haematol.2018.206540
Alvarado LJ, Huntsman HD, Cheng H, Townsley DM, Winkler T, Feng X, et al. Eltrombopag maintains human hematopoietic stem and progenitor cells under inflammatory conditions mediated by IFN-γ. Blood. 2019;133(19):2043-55. doi: 10.1182/ blood-2018-11-884486
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Imi T, Katagiri T, Hosomichi K, Zaimoku Y, Hoang Nguyen V, Nakagawa N, et al. Sustained clonal hematopoiesis by HLA-lacking hematooietic stem cells without driver mutations in aplastic anemia. Blood Adv. 2018;2(9):1000-12. doi: 10.1182/bloodadvances.2017013953
Tichelli A, de Latour RP, Passweg J, Knol-Bout C, Socié G, Marsh J, et al. Long-term outcome of a randomized controlled study in patients with newly diagnosed severe aplastic anemia treated with antithymocyte globulin and cyclosporine, with or without granulocyte colony-stimulating factor: a Severe Aplastic Anemia Working Party Trial from the European Group of Blood and Marrow Trans- plantation. Haematologica. 2020;105(5):1223-31. doi: 10.3324/haematol.2019.222562
Peffault de Latour R, Kulasekararaj A, Iacobelli S, Terwel SR, Cook R, Griffin M, et al. Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia. N Engl J Med. 2022;386(1):11-23. doi: 10.1056/NEJMoa2109965
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LiL,LiY,LinL,YinJ,XuJ,WeiJ,etal.Outcomes of allogeneic haematopoietic stem cell transplantation for patients with severe aplastic anaemia using the porcine antilymphocyte globulin-containing conditioning regimen. Ann Hematol. 2020;99(8):1863-71. doi: 10.1007/s00277-020-04111-5
Bernard F, Uppungunduri CRS, Meyer S, Cummins M, Patrick K, James B, et al. Excellent overall and chronic graft-versus-host-disease-free event-free survival in Fanconi anaemia patients undergoing matched related- and unrelated-donor bone marrow transplantation using alemtuzumab-Flu-Cy: the UK experience. Br J Haematol. 2021;193(4):804-13. doi: 10.1111/bjh.17418
Lin F, Zhang Y, Han T, Cheng Y, Mo X, Wang J, et al. A modified conditioning regimen based on low-dose cyclophosphamide and fludarabine for haploidentical hematopoietic stem cell transplant in severe aplastic anemia patients at risk of severe cardiotoxicity. Clin Transplant. 2022;36(1):e14514. doi: 10.1111/ctr.14514
Bradbury CA, Pell J, Hill Q, Bagot C, Cooper N, Ingram J, et al. Mycophenolate Mofetil for FirstLine Treatment of Immune Thrombocytopenia. N Engl J Med. 2021;385(10):885-95. doi: 10.1056/ NEJMoa2100596
WuY,YanL,WangH,LiuH,XingL,FuR,etal. Clinical study on empirical and diagnostic-driven (pre-emptive) therapy of voriconazole in severe aplastic anaemia patients with invasive fungal disease after intensive immunosuppressive therapy. Eur J Clin Microbiol Infect Dis. 2021;40(5):949-54. doi: 10.1007/s10096-020-04054-9
Jaiswal SR, Bhakuni P, Zaman S, Bansal S, Bharadwaj P, Bhargava S, et al. T cell costimulation blockade promotes transplantation tolerance in combination with sirolimus and post-transplantation cyclophosphamide for haploidentical transplantation in children with severe aplastic anemia. Transpl Immunol. 2017;43-44:54-9. doi: 10.1016/j.trim.2017.07.004
Zhao X, Feng X, Wu Z, Winkler T, Desmond R, Olnes M, et al. Persistent elevation of plasma thrombopoietin levels after treatment in severe aplastic anemia. Exp Hematol. 2018;58:39-43. doi: 10.1016/j.exphem.2017.09.006
Lee JW, Lee SE, Jung CW, Park S, Keta H, Park SK, et al. Romiplostim in patients with refractory aplastic anaemia previously treated with immunosuppressive therapy: a dose-finding and longterm treatment phase 2 trial. Lancet Haematol. 2019;6(11):e562-e572. doi: 10.1016/S2352-3026(19) 30153-X
Ise M, Iizuka H, Kamoda Y, Hirao M, Kida M, Usuki K. Romiplostim is effective for eltrombopag-refractory aplastic anemia: results of a retrospective study. Int J Hematol. 2020;112(6):787-94. doi: 10.1007/s12185-020-02971-1
Zhao LP, Sicre De Fontbrune F, Contejean A, AbrahamJ,TerriouL,ChabrotC,etal.Nationwide survey in France on the use of romiplostim in patients with refractory severe aplastic anemia. Bone Marrow Transplant. 2019;54(7):1161-3. doi: 10.1038/s41409-019-0452-1
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Young NS. Aplastic anemia. N Engl J Med. 2018; 379:1643-56. doi: 10.1056/NEJMra1413485
Putra A, Ngura A, Wiradewi, A. Characteristic overview of aplastic anemia at Sanglah Hospital, BaliIndonesia in 2017-2018. Intisari Sains Medis. 2019;10(3):497-500. doi: 10.15562/ism.v10i3.429
Filippidou M, Avgerinou G, Tsipou H, Tourkantoni N, Katsibardi K, Vlachou A, et al. Longitudinal evaluation of eltrombopag in paediatric acquired severe aplastic anaemia. Br J Haematol. 2020;190(3):e157-e59. doi: 10.1111/bjh.16766
Akram Z, Ahmed P, Kajigaya S, Satti TM, Satti HS, Chaudhary QUN, et al. Epidemiological, clinical and genetic characterization of aplastic anemia patients in Pakistan. Ann Hematol. 2019;98(2):301-12. doi: 10.1007/s00277-018-3542-z
Ally M, Magesa P, Luzzatto L. High frequency of acquired aplastic anemia in Tanzania. Am J Hematol. 2019;94(4):E86-E88. doi: 10.1002/ajh. 25388
John CO, Korubo K, Ogu R, Mmom CF, Mba AG, Chidiadi EA, et al. Management of aplastic anaemia in pregnancy in a resource poor centre. The Pan African Medical Journal. 2016;24:277. doi: 10.11604/pamj.2016.24.277.9880
Ahmed P, Chaudhry QUN, Satti TM, Mahmood SK, Ghafoor T, Shahbaz N, et al. Epidemiology of aplastic anemia: a study of 1324 cases. Hematology. 2020;25(1):48-54. doi: 10.1080/ 16078454.2019.1711344
Taj M, Shah T, Aslam SK, Zaheer S, Nawab F, Shaheen S, et al. Environmental determinants of aplastic anemia in Pakistan: a case-control study. Z Gesundh Wiss. 2016;24(5):453-60. doi: 10.1007/s10389-016-0743-6
Vaht K, Göransson M, Carlson K, Isaksson C, Lenhoff S, Sandstedt A, et al. Incidence and outcome of acquired aplastic anemia: real-world data from patients diagnosed in Sweden from 2000-2011. Haematologica. 2017;102(10):1683-90. doi: 10.3324/haematol.2017.169862
DeZernAE,ChurpekJE.Approachtothediagnosis of aplastic anemia. Blood Adv. 2021;5(12):2660-71. doi: 10.1182/bloodadvances.2021004345
Dong X, Han Y, Abeysekera IR, Shao Z, Wang H. GDF11 is increased in patients with aplastic anemia. Hematology. 2019;24(1):331-6. doi: 10.10 80/16078454.2019.1574386
Furlong E, Carter T. Aplastic anaemia: Current concepts in diagnosis and management. J Paediatr Child Health. 2020;56(7):1023-8. doi: 10.1111/jpc. 14996
LinS,HouL,LiuS,WangJ,ChenQ,ZhangB,et al. Roles of regulatory T cells in the pathogenesis of pediatric aplastic anemia. Pediatr Hematol Oncol. 2019;36(4):198-210. doi: 10.1080/08880018.2019. 1621968
Zhang J, Wu Q, Shi J, Ge M, Li X, Shao Y, et al. Involvement of interleukin-21 in the pathophysiology of aplastic anemia. Eur J Haematol. 2015;95(1):44-51. doi: 10.1111/ejh.12471
WuZ,GiudiceV,ChenJ,SunW,LinZ,Keyvanfar K, et al. Interleukin-18 plays a dispensable role in murine and likely also human bone marrow failure. Exp Hematol. 2019;69:54-64.e2. doi: 10.1016/j. exphem.2018.10.003
Ebens CL, DeFor TE, Tryon R, Wagner JE, MacMillan ML. Comparable Outcomes after HLA-Matched Sibling and Alternative Donor Hematopoietic Cell Transplantation for Children with Fanconi Anemia and Severe Aplastic Anemia. Biol Blood Marrow Transplant.2018;24(4):765-71. doi: 10.1016/j.bbmt.2017.11.031
Cooper JP, Farah RJ, Stevenson PA, Gooley TA, Storb R, Scott BL. Hematopoietic Cell Transplantation for Paroxysmal Nocturnal Hemoglobinuria in the Age of Eculizumab. Biol Blood Marrow Transplant. 2019;25(7):1331-9. doi: 10.1016/j.bbmt. 2019.01.033
Li Y, Ding S, Liu C, Chen T, Liu H, Li L, et al. Abnormalities of quantities and functions of CD56bright natural killer cells in non-severe aplastic Anemia. Hematology. 2019;24(1):405-12. doi: 10.1080/16078454.2019.1590963
Giammarco S, Peffault de Latour R, Sica S, Dufour C, Socie G, Passweg J, et al. Transplant outcome for patients with acquired aplastic anemia over the age of 40: has the outcome improved. Blood. 2018;131(17):1989-92. doi: 10.1182/blood-2017-09-807859
Camitta BM, Rappeport JM, Parkman R, Nathan DG. Selection of patients for bone marrow transplantation in severe aplastic anemia. Blood. 1975;45(3):355-63.
Killick SB, Bown N, Cavenagh J, Dokal I, Foukaneli T, Hill A, et al. Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol. 2016;172(2):187-207. doi: 10.1111/bjh.13853
Lian Y, Shi J, Nie N, Huang Z, Shao Y, Zhang J, et al. Evolution patterns of paroxysmal nocturnal hemoglobinuria clone and clinical implications in acquired bone marrow failure. Exp Hematol. 2019;77:41-50. doi: 10.1016/j.exphem.2019.08.005
Bacigalupo A. How I treat acquired aplastic anemia. Blood. 2017 Mar 16;129(11):1428-36. doi: 10.1182/blood-2016-08-693481
Dufour C, Veys P, Carraro E, Bhatnagar N, Pillon M, Wynn R, et al. Similar outcome of upfront-unrelated and matched sibling stem cell transplantation in idiopathic paediatric aplastic anaemia. A study on behalf of the UK Paediatric BMT Working Party, Paediatric Diseases Working Party and Severe Aplastic Anaemia Working Party of EBMT. Br J Haematol. 2015;171(4):585-94. doi: 10.1111/bjh.13614
Rogers ZR, Nakano TA, Olson TS, Bertuch AA, Wang W, Gillio A, et al. Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium s10.3324/haematol.2018.206540
Alvarado LJ, Huntsman HD, Cheng H, Townsley DM, Winkler T, Feng X, et al. Eltrombopag maintains human hematopoietic stem and progenitor cells under inflammatory conditions mediated by IFN-γ. Blood. 2019;133(19):2043-55. doi: 10.1182/ blood-2018-11-884486
LanY,LiuF,ChangL,LiuL,ZhangY,YiM,etal. Combination of umbilical cord mesenchymal stem cells and standard immunosuppressive regimen for pediatric patients with severe aplastic anemia. BMC Pediatr. 2021;21(1):102. doi: 10.1186/s12887021-02562-x
Winkler T, Fan X, Cooper J, Desmond R, Young DJ, Townsley DM, et al. Treatment optimization and genomic outcomes in refractory severe aplastic anemia treated with eltrombopag. Blood. 2019;133(24):2575-85. doi: 10.1182/blood.2019 000478
Imi T, Katagiri T, Hosomichi K, Zaimoku Y, Hoang Nguyen V, Nakagawa N, et al. Sustained clonal hematopoiesis by HLA-lacking hematooietic stem cells without driver mutations in aplastic anemia. Blood Adv. 2018;2(9):1000-12. doi: 10.1182/bloodadvances.2017013953
Tichelli A, de Latour RP, Passweg J, Knol-Bout C, Socié G, Marsh J, et al. Long-term outcome of a randomized controlled study in patients with newly diagnosed severe aplastic anemia treated with antithymocyte globulin and cyclosporine, with or without granulocyte colony-stimulating factor: a Severe Aplastic Anemia Working Party Trial from the European Group of Blood and Marrow Trans- plantation. Haematologica. 2020;105(5):1223-31. doi: 10.3324/haematol.2019.222562
Peffault de Latour R, Kulasekararaj A, Iacobelli S, Terwel SR, Cook R, Griffin M, et al. Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia. N Engl J Med. 2022;386(1):11-23. doi: 10.1056/NEJMoa2109965
Narita A, Muramatsu H, Ichikawa D, Hamada M, Nishikawa E, Suzuki K, et al. Relationship between plasma rabbit anti-thymocyte globulin concentration and immunosuppressive therapy response in patients with severe aplastic anemia. Eur J Haematol. 2021;107(2):255-64. doi: 10.1111/ejh.13644
Zhang F, Zhang L, Jing L, Zhou K, Wang H, Peng G, et al. High-dose cyclophosphamide compared with antithymocyte globulin for treatment of acquired severe aplastic anemia. Exp Hematol. 2013;41(4):328-34. doi: 10.1016/j.exphem.2013.01.001
LiL,LiY,LinL,YinJ,XuJ,WeiJ,etal.Outcomes of allogeneic haematopoietic stem cell transplantation for patients with severe aplastic anaemia using the porcine antilymphocyte globulin-containing conditioning regimen. Ann Hematol. 2020;99(8):1863-71. doi: 10.1007/s00277-020-04111-5
Bernard F, Uppungunduri CRS, Meyer S, Cummins M, Patrick K, James B, et al. Excellent overall and chronic graft-versus-host-disease-free event-free survival in Fanconi anaemia patients undergoing matched related- and unrelated-donor bone marrow transplantation using alemtuzumab-Flu-Cy: the UK experience. Br J Haematol. 2021;193(4):804-13. doi: 10.1111/bjh.17418
Lin F, Zhang Y, Han T, Cheng Y, Mo X, Wang J, et al. A modified conditioning regimen based on low-dose cyclophosphamide and fludarabine for haploidentical hematopoietic stem cell transplant in severe aplastic anemia patients at risk of severe cardiotoxicity. Clin Transplant. 2022;36(1):e14514. doi: 10.1111/ctr.14514
Bradbury CA, Pell J, Hill Q, Bagot C, Cooper N, Ingram J, et al. Mycophenolate Mofetil for FirstLine Treatment of Immune Thrombocytopenia. N Engl J Med. 2021;385(10):885-95. doi: 10.1056/ NEJMoa2100596
WuY,YanL,WangH,LiuH,XingL,FuR,etal. Clinical study on empirical and diagnostic-driven (pre-emptive) therapy of voriconazole in severe aplastic anaemia patients with invasive fungal disease after intensive immunosuppressive therapy. Eur J Clin Microbiol Infect Dis. 2021;40(5):949-54. doi: 10.1007/s10096-020-04054-9
Jaiswal SR, Bhakuni P, Zaman S, Bansal S, Bharadwaj P, Bhargava S, et al. T cell costimulation blockade promotes transplantation tolerance in combination with sirolimus and post-transplantation cyclophosphamide for haploidentical transplantation in children with severe aplastic anemia. Transpl Immunol. 2017;43-44:54-9. doi: 10.1016/j.trim.2017.07.004
Zhao X, Feng X, Wu Z, Winkler T, Desmond R, Olnes M, et al. Persistent elevation of plasma thrombopoietin levels after treatment in severe aplastic anemia. Exp Hematol. 2018;58:39-43. doi: 10.1016/j.exphem.2017.09.006
Lee JW, Lee SE, Jung CW, Park S, Keta H, Park SK, et al. Romiplostim in patients with refractory aplastic anaemia previously treated with immunosuppressive therapy: a dose-finding and longterm treatment phase 2 trial. Lancet Haematol. 2019;6(11):e562-e572. doi: 10.1016/S2352-3026(19) 30153-X
Ise M, Iizuka H, Kamoda Y, Hirao M, Kida M, Usuki K. Romiplostim is effective for eltrombopag-refractory aplastic anemia: results of a retrospective study. Int J Hematol. 2020;112(6):787-94. doi: 10.1007/s12185-020-02971-1
Zhao LP, Sicre De Fontbrune F, Contejean A, AbrahamJ,TerriouL,ChabrotC,etal.Nationwide survey in France on the use of romiplostim in patients with refractory severe aplastic anemia. Bone Marrow Transplant. 2019;54(7):1161-3. doi: 10.1038/s41409-019-0452-1
BentoL,BastidaJM,García-CadenasI,García-To- rres E, Rivera D, Bosch-Vilaseca A, et al. Thrombopoietin Receptor Agonists for Severe Thrombocytopenia after Allogeneic Stem Cell Transplantation: Experience of the Spanish Group of Hematopoietic Stem Cell Transplant. Biol Blood Marrow Transplant. 2019;25(9):1825-31. doi: 10.1016/j.bbmt.2019.05.023
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inmunología
tratamiento farmacológico
epidemiología
sangre
fisiopatología
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© Ciencia y Salud, 2023
Afiliaciones
Isabela María Robledo Barrios
Universidad Pontificia Bolivariana, Medellín, Colombia
Cómo citar
1.
Robledo Barrios IM. Anemia aplásica: un nuevo reto farmacológico en la práctica clínica. cysa [Internet]. 9 de marzo de 2023 [citado 19 de abril de 2024];7(1):37-46. Disponible en: https://revistas.intec.edu.do/index.php/cisa/article/view/2771