References
- Colden MA, Kumar S, Munkhbileg B, et al. Insights into the emergence of paroxysmal nocturnal hemoglobinuria. Front Immunol. 2022;12(gennaio):830172. doi:10.3389/fimmu.2021.830172
- Merck & Co. MSD manuals professional edition. s.d. Available from: https://www.msdmanuals.com/it-it/professionale. Accessed August 28, 2023.
- Hill A, DeZern AE, Kinoshita T, et al. Paroxysmal nocturnal haemoglobinuria. Nat Rev Dis Prim. 2017;3(1):17028. doi:10.1038/nrdp.2017.28
- De Fontbrune S, Flore PB, Piggin M, et al. The burden of illness of patients with paroxysmal nocturnal haemoglobinuria receiving C5 inhibitors: clinical outcomes and medical encounters from the patient perspective. Hematology. 2022;27(1):1140–1151. doi:10.1080/16078454.2022.2127630
- Shah N, Bhatt EH. Paroxysmal nocturnal hemoglobinuria. National Library of Medicine; 2023. Available from: https://www.ncbi.nlm.nih.gov/books/NBK562292/. Accessed March 27, 2024.
- Hakimi Z, Wilson K, McAughey E, et al. The cost–effectiveness, of pegcetacoplan compared with ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria, in a UK setting. J Comp Eff Res. 2022;11(13):969–985. doi:10.2217/cer-2022-0076
- Bhak RH, Mody-Patel N, Baver SB, et al. Comparative effectiveness of pegcetacoplan versus ravulizumab in patients with paroxysmal nocturnal hemoglobinuria previously treated with eculizumab: a matching-adjusted indirect comparison. Curr Med Res Opin. 2021;37(11):1913–1923. doi:10.1080/03007995.2021.1971182
- FDA approves new treatment for adults with serious rare blood disease | FDA. s.d. Consultato 4 agosto 2023. Available from: https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-new-treatment-adults-serious-rare-blood-disease. Accessed March 27, 2024.
- Hillmen P, Szer J, Weitz I, et al. Pegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2021;384(11):1028–1037. doi:10.1056/NEJMoa2029073
- Aspaveli | European medicines agency. s.d. Available from: https://www.ema.europa.eu/en/medicines/human/EPAR/aspaveli. Accessed August 4th, 2023.
- Drabo EF, Padula WV. Introduction to Markov modeling. In: David B, Logan B, William VP, editors. Handbook of Applied Health Economics in Vaccines. Oxford University Press. © Oxford University Press;2023. doi:10.1093/oso/9780192896087.003.0022
- Kulasekararaj AG, Hill A, Rottinghaus ST, et al. Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor–experienced adult patients with PNH: the 302 Study. Blood. 2019;133(6):540–549. doi:10.1182/blood-2018-09-876805
- Longworth L, Yang Y, Young T, et al. Use of generic and condition-specific measures of health-related quality of life in NICE decision-making: a systematic review, statistical modelling and survey. Health Technol Assess. 2014;18(9). doi:10.3310/hta18090
- Lloyd AJ, Gallop K, Ali S. PSY9 preference weights for quality-adjusted life-years estimation for treatments of paroxysmal nocturnal hemoglobinuria in the United Kingdom. Value Health. 2019;22(November):S902. doi:10.1016/j.jval.2019.09.2637
- Stoner KL, Harder H, Fallowfield LJ, et al. Intravenous versus subcutaneous drug administration. Which do patients prefer? A systematic review. Patient. 2015;8(2):145–153. doi:10.1007/s40271-014-0075-y
- Catalano L. Italian blood system 2021: activity data, haemovigilance and epidemiological surveillance. ISTISAN Reports, Available from: www.iss.it/documents/20126/6682486/22-25+web.pdf/192d899e-0afe-c3f7-c52df373a89097e2?t=1667829051240. Accessed March 27, 2024.