FR/UNB-P/0193 - 1. Parker, C. et al. (2005). Diagnosis and management of paroxysmal nocturnal hemoglobinuria. In Blood (Vol. 106, Issue 12, pp. 3699–3709). American Society of Hematology. https://doi.org/10.1182/blood-2005-04-1717. 2. Illingworth, A. et al. ICCS/ESCCA consensus guidelines to detect GPI-deficient cells in paroxysmal nocturnal hemoglobinuria (PNH) and related disorders part 3 - data analysis, reporting and case studies. Cytometry B Clin. Cytom. 2018;94:49–66. 3. PROTOCOLE NATIONAL DE DIAGNOSTIC ET DE SOINS APLASIES MEDULLAIRES ACQUISES ET CONSTITUTIONNELLES. (n.d.). 4. Notaro, R., & Luzzatto, L. (2022). Breakthrough Hemolysis in PNH with Proximal or Terminal Complement Inhibition. New England Journal of Medicine, 387(2), 160–166. https://doi.org/10.1056/nejmra2201664 5. Kulasekararaj, A. G. et al. (2023). Biomarkers and laboratory assessments for monitoring the treatment of patients with paroxysmal nocturnal hemoglobinuria: Differences between terminal and proximal complement inhibition. In Blood Reviews (Vol. 59). Churchill Livingstone. https://doi.org/10.1016/j.blre.2023.101041 6. Hill, A., et al. (2013). Thrombosis in paroxysmal nocturnal hemoglobinuria. Blood, The Journal of the American Society of Hematology, 121(25), 4985-4996. 7. Barcellini, W. et al. Paroxysmal Nocturnal Hemoglobinuria (Pnh): Brain Mri Ischemic Lesions In Neurologically Asymtomatic Patients. Sci Rep 8, 476 (2018). https://doi.org/10.1038/s41598- 017-18936-0. 8. Wagner-Ballon, O., et al. (2024). High Proportion of PNH Type II Neutrophils, Ie Relative Percentage≥ 3%, Is Associated with Thrombosis in Patients Displaying a PNH Clone> 1%: Evidence from Analysis of the 5-Year French Nation-Wide Multicenter Observational Study. Blood, 144, 4080. https://doi.org/10.1182/blood-2024-204647.

FR/UNB-P/0200 - 1. Parker, C. et al., (2005). Diagnosis and management of paroxysmal nocturnal hemoglobinuria. In Blood (Vol. 106, Issue 12, pp. 3699–3709). American Society of Hematology. https://doi.org/10.1182/blood-2005-04-1717 2. Notaro, R., & Luzzatto, L. (2022). Breakthrough Hemolysis in PNH with Proximal or Terminal Complement Inhibition. New England Journal of Medicine, 387(2), 160–166. https://doi.org/10.1056/nejmra2201664 3. PROTOCOLE NATIONAL DE DIAGNOSTIC ET DE SOINS APLASIES MEDULLAIRES ACQUISES ET CONSTITUTIONNELLES. (n.d.). 4. Kulasekararaj, A. G. et al., (2023). Biomarkers and laboratory assessments for monitoring the treatment of patients with paroxysmal nocturnal hemoglobinuria: Differences between terminal and proximal complement inhibition. In Blood Reviews (Vol. 59). Churchill Livingstone. https://doi.org/10.1016/j.blre.2023.101041 5. Hill A, et al., (2013). Thrombosis in paroxysmal nocturnal hemoglobinuria. Blood. 21: 4985-4996. 6. Kulasekararaj, A. G. et al., (2021). Monitoring of patients with paroxysmal nocturnal hemoglobinuria on a complement inhibitor. In American Journal of Hematology (Vol. 96, Issue 7, pp. E232–E235). John Wiley and Sons Inc. https://doi.org/10.1002/ajh.26176 7. Hémoglobinurie paroxystique nocturne ©Orphanet Focus Handicap / Encyclopédie Orphanet du Handicap Hémoglobinurie paroxystique nocturne Synonymes : HPN / Maladie de MarchiafavaMicheli Encyclopédie Orphanet du Handicap. (n.d.). www.orpha.net/data/patho/Han/Int/fr/HemoglobinurieParoxystiqueNocturne_FR_fr_HAN_ORP HA447.pdf 8. Fattizzo, B. et al., (2022). Managing Fatigue in Patients with Paroxysmal Nocturnal Hemoglobinuria: A Patient-Focused Perspective. In Journal of Blood Medicine (Vol. 13, pp. 327– 335). Dove Medical Press Ltd. https://doi.org/10.2147/JBM.S339660 9. Kelly, R. J. et al., (2024) Treatment outcomes of complement protein C5 inhibition in 509 UK patients with paroxysmal nocturnal hemoglobinuria. In Blood (Vol. 143, Issue 12, pp. 1157-1166. https://doi.org/10.1182/blood.2023021762 10.1182/blood.2023021762 10. Ricklin, D. et al., (2017). The renaissance of complement therapeutics. In Nature Reviews Nephrology (Vol. 14, Issue 1, pp. 26–47). Nature Publishing Group. 11. Avis du Haut Conseil de la Santé Publique en vigueur.

FR/UNB-P/0159 – Juillet 2024 Bibliographie : - Cançado, R. D., Araújo, A. da S., Sandes, A. F., Arrais, C., Lobo, C. L. de C., Figueiredo, M. S., Gualandro, S. F. M., Saad, S. T. O., & Costa, F. F. (2021). Consensus statement for diagnosis and treatment of paroxysmal nocturnal haemoglobinuria. Hematology, Transfusion and Cell Therapy, 43(3), 341–348. https://doi.org/10.1016/j.htct.2020.06.006 - De Latour, R. P., Mary, J. Y., Salanoubat, C., Terriou, L., Etienne, G., Mohty, M., Roth, S., De Guibert, S., Maury, S., Cahn, J. Y., & Socie, G. (2008). Paroxysmal nocturnal hemoglobinuria: Natural history of disease subcategories. Blood, 112(8), 3099–3106. https://doi.org/10.1182/blood-2008-01-133918 - Haute Autorité de Santé. (2023). PROTOCOLE NATIONAL DE DIAGNOSTIC ET DE SOINS APLASIES MEDULLAIRES ACQUISES ET CONSTITUTIONNELLES. - Höchsmann, B., Peffault de Latour, R., Hill, A., Röth, A., Devos, T., Patriquin, C. J., Chou, W. C., Jain, D., Zu, K., Wu, C., & Lee, J. W. (2023). Risk factors for thromboembolic events in patients with paroxysmal nocturnal hemoglobinuria (PNH): a nested case–control study in the International PNH Registry. Annals of Hematology, 102(11), 2979–2988. https://doi.org/10.1007/s00277-023-05402-3 - Kokoris, S. I., Gavriilaki, E., Miari, A., Travlou, Α., Kyriakou, E., Anagnostopoulos, A., & Grouzi, E. (2018). Renal involvement in paroxysmal nocturnal hemoglobinuria: an update on clinical features, pathophysiology and treatment. Hematology, 23(8), 558–566. https://doi.org/10.1080/10245332.2018.1444563 - Kulasekararaj, A. G., Brodsky, R. A., & Hill, A. (2021). Monitoring of patients with paroxysmal nocturnal hemoglobinuria on a complement inhibitor. In American Journal of Hematology (Vol. 96, Issue 7, pp. E232–E235). John Wiley and Sons Inc. https://doi.org/10.1002/ajh.26176 - Orphanet. (2019). Hémoglobinurie paroxystique nocturne. www.orpha.net/data/patho/Han/Int/fr/HemoglobinurieParoxystiqueNocturne_FR_fr_HAN_ORPHA447.pdf - Parker, C., Omine, M., Richards, S., Nishimura, J. I., Bessler, M., Ware, R., Hillmen, P., Luzzatto, L., Young, N., Kinoshita, T., Rosse, W., & Socié, G. (2005). Diagnosis and management of paroxysmal nocturnal hemoglobinuria. In Blood (Vol. 106, Issue 12, pp. 3699–3709). American Society of Hematology. https://doi.org/10.1182/blood-2005-04-1717 - PROTOCOLE NATIONAL DE DIAGNOSTIC ET DE SOINS APLASIES MEDULLAIRES ACQUISES ET CONSTITUTIONNELLES. (n.d.).