Stable responses to danicopan as add-on to ravulizumab in two patients with paroxysmal nocturnal hemoglobinuria
Author:
Funder
Medical University of Vienna
Publisher
Springer Science and Business Media LLC
Link
https://link.springer.com/content/pdf/10.1007/s00277-024-05858-x.pdf
Reference9 articles.
1. Risitano AM, Notaro R, Marando L, Serio B, Ranaldi D, Seneca E, Ricci P, Alfinito F, Camera A, Gianfaldoni G, Amendola A, Boschetti C, Di Bona E, Fratellanza G, Barbano F, Rodeghiero F, Zanella A, Iori AP, Selleri C, Luzzatto L, Rotoli B (2009) Complement fraction 3 binding on erythrocytes as additional mechanism of disease in paroxysmal nocturnal hemoglobinuria patients treated by eculizumab. Blood 113:4094–4100. https://doi.org/10.1182/blood-2008-11-189944
2. Kulasekararaj AG, Risitano AM, Maciejewski JP, Notaro R, Browett P, Lee JW, Huang M, Geffner M, Brodsky RA (2021) Phase 2 study of danicopan in patients with paroxysmal nocturnal hemoglobinuria with an inadequate response to eculizumab. Blood 138:1928–1938. https://doi.org/10.1182/blood.2021011388
3. Lee JW, Griffin M, Kim JS, Lee Lee LW, Piatek C, Nishimura JI, Carrillo Infante C, Jain D, Liu P, Filippov G, Sicre de Fontbrune F, Risitano A, Kulasekararaj AG, ALXN2040-PNH-301 Investigators (2023) Addition of danicopan to ravulizumab or eculizumab in patients with paroxysmal nocturnal haemoglobinuria and clinically significant extravascular haemolysis (ALPHA): a double-blind, randomised, phase 3 trial. Lancet Haematol 10(12):e955–e965. https://doi.org/10.1016/S2352-3026(23)00315-0
4. Gurnari C, Awada H, Pagliuca S, Dima D, Ullah F, Kawashima N, Kubota Y, Colak C, Visconte V, Patel BJ, Dhillon V, Marneni N, Balasubramanian SK, Kishtagari A, Bat T, Maciejewski JP (2024) Paroxysmal nocturnal hemoglobinuria-related thrombosis in the era of novel therapies: a 2043 patient/years analysis. Blood Mar 21:blood.2024023988. https://doi.org/10.1182/blood.2024023988
5. Notaro R, Luzzatto L (2022) Breakthrough hemolysis in PNH with proximal or terminal complement inhibition. N Engl J Med 387(2):160–166. https://doi.org/10.1056/NEJMra2201664
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