Bibliografía

(1) Brodsky, R. A. (2014). Paroxysmal nocturnal hemoglobinuria. Blood, 124(18), 2804–2811. https://doi.org

(2) Rother, R. P., Rollins, S. A., Mojcik, C. F., Brodsky, R. A., & Bell, L. (2007). Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Nature Biotechnology, 25(11), 1256–1264. https://doi.org

(3) Sharma, V. R. (2013). Paroxysmal nocturnal hemoglobinuria: Pathogenesis, testing, and diagnosis. Clinical Advances in Hematology & Oncology, 11(9 Suppl. 13), 2–8.

(4) Hillmen, P., Lewis, S. M., Bessler, M., Luzzatto, L., & Dacie, J. V. (1995). Natural history of paroxysmal nocturnal hemoglobinuria. The New England Journal of Medicine, 333(19), 1253–1258. https://doi.org

(5) Hill, A., Kelly, R. J., & Hillmen, P. (2013). Thrombosis in paroxysmal nocturnal hemoglobinuria. Blood, 121(25), 4985–4996. https://doi.org

(6) Villegas, A., & Gaya, A. (Coords.). (s. f.). Consenso español para el diagnóstico y tratamiento de la hemoglobinuria paroxística nocturna. Sociedad Española de Hematología y Hemoterapia. https://sehh.es

(7) Richards, S. J., Painter, D., Dickison, A., Griffin, M., Munir, T., Arnold, L., Dyer, M. J. S., McMullin, M. F., Kulasekararaj, A., Hill, A., & Hillmen, P. (2021). 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. European Journal of Haematology, 107(2), 211–218. https://doi.org

(8) Panse, J. (2023). Paroxysmal nocturnal hemoglobinuria: Where we stand. American Journal of Hematology, 98(Suppl. 4), S20–S32. https://doi.org

(9) Villegas, A., Arrizabalaga, B., Bonanad, S., Colado, E., Gaya, A., González, A., García-Marco, J. A., Hernández-Navarro, F., Jarque, I., Raya, J. M., Sánchez-Guijo, F., & Grupo de Trabajo de HPN de la Sociedad Española de Hematología y Hemoterapia. (2016). Consenso español para el diagnóstico y tratamiento de la hemoglobinuria paroxística nocturna. Medicina Clínica, 146(6), 271–278. https://doi.org

(10) Hillmen, P., Elebute, M., Kelly, R., Urbano-Ispizua, A., Hill, A., Rother, R. P., Khursigara, G., Fu, C.-L., Maciejewski, J. P., & Brodsky, R. A. (2010). Long-term effect of the complement inhibitor eculizumab on kidney function in patients with paroxysmal nocturnal hemoglobinuria. American Journal of Hematology, 85(8), 553–559. https://doi.org

(11) Villegas, A., Núñez, R., Gaya, A., Cuevas-Ruiz, M. V., Bosch, J. M., Carral, A., Jarque, I., Raya, J. M., Bonanad, S., Arrizabalaga, B., Colado, E., González, A., Sánchez-Guijo, F., García-Marco, J. A., Hernández-Navarro, F., & Grupo Español de HPN. (2017). Presence of acute and chronic renal failure in patients with paroxysmal nocturnal hemoglobinuria: Results of a retrospective analysis from the Spanish PNH Registry. Annals of Hematology, 96(10), 1727–1733. https://doi.org

(12) Hill, A., Sapsford, R. J., Scally, A., Kelly, R., Richards, S. J., Khursigara, G., McMullin, M. F., Muus, P., Röth, A., Hillmen, P., & Brodsky, R. A. (2012). Under-recognized complications in patients with paroxysmal nocturnal haemoglobinuria: Raised pulmonary pressure and reduced right ventricular function. British Journal of Haematology, 158(3), 409–414. https://doi.org

(13) Illingworth, A. J., Marinov, I., & Sutherland, D. R. (2019). Sensitive and accurate identification of PNH clones based on ICCS/ESCCA PNH consensus guidelines—A summary. International Journal of Laboratory Hematology, 41(Suppl. 1), 73–81. https://doi.org

(14) Borowitz, M. J., Craig, F. E., DiGiuseppe, J. A., Illingworth, A. J., Rosse, W., Sutherland, D. R., Wittwer, C. T., Richards, S. J., & Dahlback, B. (2010). Guidelines for the diagnosis and monitoring of paroxysmal nocturnal hemoglobinuria and related disorders by flow cytometry. Cytometry Part B: Clinical Cytometry, 78B(4), 211–230. https://doi.org

(15) Young, N. S., Calado, R. T., & Scheinberg, P. (2006). Current concepts in the pathophysiology and treatment of aplastic anemia. Blood, 108(8), 2509–2519. https://doi.org

(16) Hillmen, P., Young, N. S., Schubert, J., Brodsky, R. A., Socié, G., Muus, P., Röth, A., Szer, J., Elebute, M. O., Nakamura, R., Browne, P., Risitano, A. M., Hill, A., Schrezenmeier, H., Fu, C.-L., & Maciejewski, J. P. (2006). The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. The New England Journal of Medicine, 355(12), 1233–1243. https://doi.org

(17) Brodsky, R. A., Young, N. S., Antonioli, E., Risitano, A. M., Schrezenmeier, H., Schubert, J., Gaya, A., Coyle, L., de Castro, C., Fu, C.-L., Maciejewski, J. P., Bessler, M., Kroon, H. A., Rother, R. P., Hillmen, P., & the PNH Study Group. (2008). Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood, 111(4), 1840–1847. https://doi.org

(18) Lee, J. W., Sicre de Fontbrune, F., Wong Lee Lee, L., Pessoa, V., Gualandro, S., Füreder, W., Ptushkin, V., Rottinghaus, S. T., Volles, L., & Peffault de Latour, R. (2019). Ravulizumab (ALXN1210) versus eculizumab in adult patients with PNH naive to complement inhibitors: The 301 study. Blood, 133(6), 530–539. https://doi.org

(19) Kulasekararaj, A. G., Hill, A., Langemeijer, S., Wells, R., González Fernández, F. A., Gaya, A., Lee, J. W., Gutierrez, E. O., de Fontbrune, F. S., Szer, J., Nishimura, J. I., Schrezenmeier, H., Röth, A., Volles, L., Damokosh, A. I., & Peffault de Latour, R. (2021). One-year outcomes from a phase 3 randomized trial of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria who received prior eculizumab. European Journal of Haematology, 106(3), 389–397. https://doi.org

(20) Kulasekararaj, A., Brodsky, R., Schrezenmeier, H., Griffin, M., Röth, A., Piatek, C., Lee, J. W., Patriquin, C., Nishimura, J. I., Volles, L., Damokosh, A., & Peffault de Latour, R. (2025). Ravulizumab demonstrates long-term efficacy, safety and favorable patient survival in patients with paroxysmal nocturnal hemoglobinuria. Annals of Hematology, 104(1), 81–94. https://doi.org

(21) Kulasekararaj, A. G., Risitano, A. M., Maciejewski, J. P., Notaro, R., Browett, P., Lee, J. W., Cacace, F., de Castro, C., Szer, J., Nishimura, J. I., Peffault de Latour, R., Patriquin, C. J., Griffin, M., Hill, A., Volles, L., & Brodsky, R. A. (2021). Phase 2 study of danicopan in patients with paroxysmal nocturnal hemoglobinuria with an inadequate response to eculizumab. Blood, 138(20), 1928–1938. https://doi.org

(22) Risitano, A. M., Kulasekararaj, A. G., Lee, J. W., Maciejewski, J. P., Notaro, R., Brodsky, R. A., Szer, J., Browett, P., de Castro, C., Peffault de Latour, R., Nishimura, J. I., Griffin, M., Hill, A., Volles, L., & Patriquin, C. J. (2021). Danicopan: An oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria. Haematologica, 106(12), 3188–3197. https://doi.org

(23) Kulasekararaj, A., Griffin, M., Piatek, C., Shammo, J., Nishimura, J. I., Patriquin, C., Lee, J. W., Röth, A., Schrezenmeier, H., Brodsky, R. A., Volles, L., Damokosh, A., Hill, A., & Peffault de Latour, R. (2025). Long-term efficacy and safety of danicopan as add-on therapy to ravulizumab or eculizumab in PNH with significant extravascular hemolysis. Blood, 145(8), 811–822. https://doi.org

(24) Peffault de Latour, R., Röth, A., Kulasekararaj, A., Weitz, I. C., Nishimura, J. I., Sicre de Fontbrune, F., Kim, J. S., Lee, J. W., Patriquin, C. J., Schrezenmeier, H., Volles, L., Damokosh, A., & Risitano, A. M. (2025). Oral iptacopan monotherapy in paroxysmal nocturnal haemoglobinuria: Final 48-week results from the open-label, randomised, phase 3 APPLY-PNH trial in anti-C5-treated patients and the open-label, single-arm, phase 3 APPOINT-PNH trial in patients previously untreated with complement inhibitors. The Lancet Haematology, 12(7), e508–e520. https://doi.org

(25) Röth, A., Peffault de Latour, R., Kulasekararaj, A., Weitz, I. C., Nishimura, J. I., Sicre de Fontbrune, F., Kim, J. S., Lee, J. W., Patriquin, C. J., Schrezenmeier, H., Risitano, A. M., Volles, L., Damokosh, A., & APPLYPNH Investigators. (2024). Iptacopan versus standard of care in patients with paroxysmal nocturnal hemoglobinuria and residual anemia despite anti-C5 therapy (APPLY-PNH). The New England Journal of Medicine, 390(6), 552–564. https://doi.org

(26) Peffault de Latour, R., Röth, A., Risitano, A. M., Weitz, I. C., Nishimura, J. I., Kim, J. S., Lee, J. W., Patriquin, C. J., Schrezenmeier, H., Sicre de Fontbrune, F., Volles, L., Damokosh, A., & APPOINT-PNH Investigators. (2024). Iptacopan monotherapy in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria (APPOINT-PNH). The New England Journal of Medicine, 390(6), 566–576. https://doi.org