im MÉDICO | 59.2 26 1. Estrategia en Enfermedades Raras del Sistema Nacional de Salud. 2009. Consultado en: https://www.mscbs.gob.es/ organizacion/sns/planCalidadSNS/docs/enfermedadesRaras.pdf 2. IFPMA (2017). RARE DISEASES: shaping a future with no-one left behind. Available at: https://www.ifpma.org/ wp-content/uploads/2017/02/IFPMA_Rare_Diseases_ Brochure_28Feb2017_FINAL.pdf Last accessed Feb. 2022 3. Orphanet. Paroxysmal Nocturnal Hemoglobinuria. [citado 2014 Aug 26]. Disponible en http://www.orpha.net/consor/ cgibin/OC_Exp.php?lng=ES&Expert=447. 4. Hill A, Richards SJ, Hillmen P. Recent developments in the understanding and management of paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2007 May;137(3):181-92. 5. Socié G, Mary J, de Gramont A, Rio B, Leporrier M, Rose C et al. Paroxysmal nocturnal haemoglobinuria: long-term followup and prognostic factors. The Lancet. 1996;348(9027):573577. 6. Sahin F, et al. Am J Blood Res. 2016;6(2):19-27. 7. Peacock-Young B, Macrae FL, Newton DJ, Hill A, Ariëns RA. The prothrombotic state in paroxysmal nocturnal hemoglobinuria: A multifaceted source. Haematologica. 2017;103(1):9–17. 8. Borowitz MJ, Craig FE, DiGiuseppe JA et al; for Clinical Cytometry Society Guidelines for the diagnosis and monitoring of paroxysmal nocturnal hemoglobinuria and related disorders by flow cytometry. Cytometry B Clin Cytom. 2010;78B:211-230. 9. Parker CJ. Management of paroxysmal nocturnal hemoglobinuria in the era of complement inhibitory therapy. Hematology Am Soc Hematol Educ Program.2011;2011:21-29. 10. Parker CJ. Update on the diagnosis and management of paroxysmal nocturnal hemoglobinuria. Hematology Am Soc Hematol Educ Program. 2016;2016:208-216. 11. Luzzatto L, Bessler M, Rotoli B. Somatic mutations in paroxysmal nocturnal hemoglobinuria: A blessing in disguise? Cell. 1997;88(1):1–4. 12. DeZern AE and Brodsky RA. Paroxysmal Nocturnal Hemoglobinuria. A Complement-Mediated Hemolytic Anemia. Hematol Oncol Clin North Am. 2015 Jun; 29(3):479–494. 13. Atkinson, John P., et al. Sistema del complemento humano: conceptos básicos e importancia clínica. Inmunología Clínica: Principios Y Práctica (2019): 299. 14. Weitz I, Meyers G, Lamy T, Cahn J, Uranga M, García Vela J et al. Cross-sectional validation study of patient-reported outcomes in patients with paroxysmal nocturnal haemoglobinuria. Internal Medicine Journal. 2013;43(3):298-307. 15. Lee JW, et al. Clinical signs and symptoms associated with increased risk for thrombosis in patients with paroxysmal nocturnal hemoglobinuria from a Korean Registry. Int J Hematol. 2013;97(6):749–57. 16. Dacie JV, Lewis SM. Paroxysmal nocturnal haemoglobinuria: clinical manifestations, haematology, and nature of the disease. Ser Haemat. 1972;5:3-23. 17. Nishimura J, Kanakura Y, Ware R, Shichishima T, Nakakuma H, Ninomiya H et al. Clinical Course and Flow Cytometric Analysis of Paroxysmal Nocturnal Hemoglobinuria in the United States and Japan. Medicine. 2004;83(3):193-207. 18. Lee J, et al. Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study. Blood. 2019;133(6):530-539. 19. Kulasekararaj A, et al. Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor–experienced adult patients with PNH: the 302 study. Blood. 2019;133(6):540-549. 20. Hillmen P., et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007;110(12):4123-4128. 21. Morado M, Freire Sandes A, Colado E, Subirá D, Isusi P, Soledad Noya M, et al. Diagnostic screening of paroxysmal nocturnal hemoglobinuria: Prospective multicentric evaluation of the current medical indications. Cytometry Part B: Clinical Cytometry. 2016;92(5):361–70. 22. Parker C. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood. 2005;106(12):3699–709. 23. Kelly R, Hill A, Arnold L, Brooksbank G, Richards S, Cullen M et al. Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood. 2011;117(25):6786-6792. 24. Hillmen, P., et al. (1995). Natural history of paroxysmal nocturnal hemoglobinuria. The New England journal of medicine, 333(19), 1253–1258. 25. Ficha técnica Ultomiris®. Alexion Europe SAS. Septiembre 2021. Disponible en: https://www.podcastshua.com/bibliotecas/content/7969a2da-8614-4635-8b07-522876469598. pdf ES/ULT-P/0110 Febrero 2023 Referencias:
RkJQdWJsaXNoZXIy NTI5ODA=