Bibliografía

(1) Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014;124(18):2804-2811. doi: 10.1182/blood-2014-02-522128. Epub 2014 Sep 18. PMID: 25237200; PMCID: PMC4215311.
(2) Rother RP, Rollins SA, Mojcik CF, Brodsky RA, Bell L. Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Nat Biotechnol. 2007 Nov;25(11):1256-1264. doi: 10.1038/nbt1344. Erratum in: Nat Biotechnol. 2007 Dec;25(12):1488. PMID: 17989688.
(3) Sharma VR. Paroxysmal nocturnal hemoglobinuria: pathogenesis, testing, and diagnosis. Clin Adv Hematol Oncol. 2013;11 Suppl 13(9):2-8. PMID: 25856267
(4) Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med. 1995;333(19):1253-1258. doi: 10.1056/NEJM199511093331904. PMID: 7566002.
(5) Hill A, Kelly RJ, Hillmen P. Thrombosis in paroxysmal nocturnal hemoglobinuria. Blood. 2013;121(25):4985-4996; quiz 5105. doi: 10.1182/blood-2012-09-311381. Epub 2013 Apr 22. PMID: 23610373.
(6) Guía Clínica HPN. Consenso Español para el diagnóstico y el Tratamiento de la Hemoglobinuria Paroxística Nocturna. Coordinadoras Ana Villegas, Anna Gaya. Disponible en https://www.sehh.es/publicaciones/guías-recomendaciones/guía-clinica-en-hpn
(7) Richards SJ, Painter D, Dickison A,Griffin M, Munir T, Arnold L et al. The incidence and prevalence of patients with paroxysmal nocturnal haemoglobinuria and aplastic anaemia PNH syndrome: A retrospective analysis of the UK’s population-based haematological malignancy research network 2004- 2018. Eur J Haematol. 2021;107(2):211-218. PMID: 34060690.
(8) Panse J. Paroxysmal nocturnal hemoglobinuria: Where we stand. Am J Hematol. 2023;98 Suppl 4:S20-S32. PMID: 36594182.
(9) Villegas A, Arrizabalaga B, Bonanad S, Colado E, Gaya A, González A et al.; Grupo de Trabajo de HPN de la Sociedad Española de Hematología y Hemoterapia. Consenso español para el diagnóstico y tratamiento de la Hemoglobinuria Paroxística Nocturna. Med Clin (Barc). 2016;146(6):271-278. PMID: 26895645.
(10) Hillmen P, Elebute M, Kelly R, Urbano-Ispizua A, Hill A, Rother RP, et al. Long-term effect of the complement inhibitor eculizumab on kidney function in patients with paroxysmal nocturnal hemoglobinuria. Am J Hematol. 2010;85(8):553-559. doi: 10.1002/ajh.21757. Erratum in: Am J Hematol. 2010 ;85(11):911. PMID: 20658586.
(11) Villegas A, Núñez R, Gaya A, Cuevas-Ruiz MV, Bosch JM, Carral A, et al. Presence of acute and chronic renal failure in patients with paroxysmal nocturnal hemoglobinuria: results of a retrospective analysis from the Spanish PNH Registry. Ann Hematol. 2017;96(10):1727-1733. doi: 10.1007/s00277-017-3059-x. Epub 2017 Jul 26. PMID: 28748287.
(12) Hill A, Sapsford RJ, Scally A, Kelly R, Richards SJ, Khurisgara G, et al. Under-recognized complications in patients with paroxysmal nocturnal haemoglobinuria: raised pulmonary pressure and reduced right ventricular function. Br J Haematol. 2012;158(3):409-414. PMID: 22639982.
(13) Illingworth AJ, Marinov I, Sutherland DR. Sensitive and accurate identification of PNH clones based on ICCS/ESCCA PNH Consensus Guidelines-A summary. Int J Lab Hematol. 2019;41 Suppl 1:73-81. PMID: 31069981.
(14) Borowitz MJ, Craig FE, Digiuseppe JA, Illingworth AJ, Rosse W, Sutherland DR et al . Guidelines for the diagnosis and monitoring of paroxysmal nocturnal hemoglobinuria and related disorders by flow cytometry. Cytometry B Clin Cytom. 2010;78(4):211-230. Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood. 2006;108(8):2509-2519. doi: 10.1182/blood-2006-03-010777. Epub 2006 Jun 15. PMID: 16778145;PMCID: PMC1895575.
(15) Hillmen P, Young NS, Schubert J, Brodsky RA, Socié G, Muus P, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglo¬binuria. N Engl J Med. 2006;355:1233-1243.
(16) Brodsky RA, Young NS, Antonioli E, Risitano AM, Schrezenmeier H, Schubert J, et al. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood. 2008;111:1840-1847.
(17) Lee JW, Sicre de Fontbrune F, Wong Lee Lee L, Pessoa V, Gualandro S, Füreder W, et al. Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study. Blood. 2019;133:530-539.
(18) Kulasekararaj AG, Hill A, Langemeijer S, Wells R, González Fernández FA, Gaya A, et al. One-year outcomes from a phase 3 randomized trial of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria who received prior eculizumab. Eur J Haematol. 2021;106:389-397.
(19) Kulasekararaj A, Brodsky R, Schrezenmeier H, Griffin M, Röth A, Piatek C, et al. Ravulizumab demonstrates long-term efficacy, safety and favorable patient survival in patients with paroxysmal nocturnal hemoglobinuria. Ann Hematol. 2025;104(1):81-94. doi: 10.1007/s00277- 025-06193-5. Epub 2025 Jan 22. PMID: 39841198; PMCID: PMC11868214.
(20) Kulasekararaj AG, Risitano AM, Maciejewski JP, Notaro R, Browett P, Lee JW, et al. Phase 2 study of danicopan in patients with paroxysmal nocturnal hemoglobinuria with an inadequate response to eculizumab. Blood. 2021;138:1928-1938.
(21) Risitano AM, Kulasekararaj AG, Lee JW, Maciejewski JP, Notaro R, Brodsky R, et al. Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria. Haematologica. 2021;106:3188-3197.
(22) Kulasekararaj A, Griffin M, Piatek C, Shammo J, Nishimura JI, Patriquin C, et al. Long-term efficacy and safety of danicopan as add-on therapy to ravulizumab or eculizumab in PNH with significant EVH. Blood. 2025;145(8):811-822. doi: 10.1182/blood.2024026299. PMID: 39700502; PMCID:PMC11867134.
(23) Villegas Martínez, A., & Gaya Valls, A. (Coords.). (2023). Consenso español para el diagnóstico y el tratamiento de la hemoglobinuria paroxística nocturna: Guía clínica. Fundación Española de Hematología y Hemoterapia-Sociedad Española de Hematología y Hemoterapia. https://www.sehh.es/publicaciones/guias-recomendaciones/guia-clinica-en-hpn