Page 177 - 2021_12-Haematologica-web
P. 177
Danicopan, an oral factor D inhibitor for PNH
regulatory protein, decay accelerating fac- tor. Proc Natl Acad Sci U S A. 1983;80(16):5066-5070.
8. Holguin MH, Fredrick LR, Bernshaw NJ, Wilcox LA, Parker CJ. Isolation and charac- terization of a membrane protein from nor- mal human erythrocytes that inhibits reac- tive lysis of the erythrocytes of paroxysmal nocturnal hemoglobinuria. J Clin Invest. 1989;84(1):7-17.
9. Lachmann PJ, Halbwachs L. The influence of C3b inactivator (KAF) concentration on the ability of serum to support complement activation. Clin Exp Immunol. 1975; 21(1):109-114.
10. Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006;355(12):1233-1243.
11. Brodsky RA, Young NS, Antonioli E, et al. Multicenter phase 3 study of the comple- ment inhibitor eculizumab for the treat- ment of patients with paroxysmal noctur- nal hemoglobinuria. Blood. 2008; 111(4): 1840-1847.
12. Hillmen P, Muus P, Dührsen U, et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with parox- ysmal nocturnal hemoglobinuria. Blood. 2007;110(12):4123-4128.
13. Kelly RJ, Hill A, Arnold LM, et al. Long- term treatment with eculizumab in parox- ysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood. 2011;117(25):6786-6792.
14. Loschi M, Porcher R, Barraco F, et al. Impact of eculizumab treatment on paroxysmal nocturnal hemoglobinuria: a treatment ver- sus no-treatment study. Am J Hematol. 2016;91(4):366-370.
15. Lee JW, Sicre de Fontbrune F, Wong Lee Lee L, et al. Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study. Blood. 2019;133(6):530-539.
16. 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.
17. Risitano AM, Notaro R, Marando L, et al. Complement fraction 3 binding on erythro-
cytes as additional mechanism of disease in paroxysmal nocturnal hemoglobinuria patients treated by eculizumab. Blood. 2009;113(17):4094-4100.
18. Hill A, Rother RP, Arnold L, et al. Eculizumab prevents intravascular hemoly- sis in patients with paroxysmal nocturnal hemoglobinuria and unmasks low-level extravascular hemolysis occurring through C3 opsonization. Haematologica. 2010; 95(4):567-573.
19. Luzzatto L, Risitano AM, Notaro R. Paroxysmal nocturnal hemoglobinuria and eculizumab. Haematologica. 2010; 95(4): 523-526.
20. Risitano AM, Notaro R, Luzzatto L, Hill A, Kelly R, Hillmen P. Paroxysmal nocturnal hemoglobinuria--hemolysis before and after eculizumab. N Engl J Med. 2010; 363(23):2270-2272.
21. Notaro R, Sica M. C3-mediated extravascu- lar hemolysis in PNH on eculizumab: mechanism and clinical implications. Semin Hematol. 2018;55(3):130-135.
22. Risitano AM, Marotta S. Toward comple- ment inhibition 2.0: Next generation anti- complement agents for paroxysmal noctur- nal hemoglobinuria. Am J Hematol. 2018; 93(4):564-577.
23. Taylor RP, Lindorfer MA. Mechanisms of complement-mediated damage in hemato- logical disorders. Semin Hematol. 2018; 55(3):118-123.
24. Yuan X, Gavriilaki E, Thanassi JA, et al. Small-molecule factor D inhibitors selec- tively block the alternative pathway of complement in paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. Haematologica. 2017; 102(3):466-475.
25. Harboe M, Ulvund G, Vien L, Fung M, Mollnes TE. The quantitative role of alter- native pathway amplification in classical pathway induced terminal complement activation. Clin Exp Immunol. 2004; 138(3): 439-446.
26. Wiles JA, Galvan MD, Podos SD, Geffner M, Huang M. Discovery and development of the oral complement factor D inhibitor danicopan (ACH-4471). Curr Med Chem. 2020;27(25):4165-4180.
27. Sprong T, Roos D, Weemaes C, et al.
28.
Deficient alternative complement pathway activation due to factor D deficiency by 2 novel mutations in the complement factor D gene in a family with meningococcal infections. Blood. 2006;107(12):4865-4870. Granoff DM, Kim H, Topaz N, MacNeil J, Wang X, McNamara LA. Differential effects of therapeutic complement inhibitors on serum bactericidal activity against non-groupable meningococcal iso- lates recovered from patients treated with eculizumab. Haematologica. 2019; 104(8): e340-e344.
29. Konar M, Granoff DM. Eculizumab treat- ment and impaired opsonophagocytic killing of meningococci by whole blood from immunized adults. Blood. 2017; 130(7):891-899.
30.
31.
32.
33.
34.
Kulasekararaj A, Risitano AM, Maciejewski JP, et al. A phase 2 open-label study of dan- icopan (ACH-0144471) in patients with paroxysmal nocturnal hemoglobinuria (PNH) who have an inadequate response to eculizumab monotherapy. Blood. 2019; 134(Suppl_1):3514.
Risitano AM, Marotta S, Ricci P, et al. Anti- complement treatment for paroxysmal nocturnal hemoglobinuria: time for proxi- mal complement inhibition? A position paper from the SAAWP of the EBMT. Front Immunol. 2019;10:1157.
Risitano AM, Roth A, Soret J, et al. LNP023 - a new oral complement factor-B inhibitor normalizes hemoglobin in paroxysmal noc- turnal hemoglobinuria patients with poor response to eculizumab, both as add-on and monotherapy. The 46th Annual Meeting of the European Society for Blood and Marrow Transplantation; August 29 - September 1, 2020; O019.
Hillmen P, Szer J, Weitz I, et al. Results of the Pegasus phase III randomized trial demonstrating superiority of the C3 inhibitor, pegcetacoplan, compared to eculizumab in patients with paroxysmal nocturnal hemoglobinuria. The 25th European Hematology Association Annual Congress; June 12, 2020; 295012; S192. Holers VM. The complement system as a therapeutic target in autoimmunity. Clin Immunol. 2003;107(3):140-151.
haematologica | 2021; 106(12)
3197