[1] Rother RP, Rollins SA, Mojcik CF, et al. Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria[J]. Nat Biotechnol, 2007, 25(11):1256-1264. [2] Parker CJ. The pathophysiology of paroxysmal nocturnal hemoglobinuria[J]. Exp Hematol, 2007, 35 (4): 523-533. [3] Bessler M, Hiken J. The pathophysiology of disease in patients with paroxysmal nocturnal hemoglobinuria[J]. Hematology Am Soc Hematol Educ Program, 2008, 2008:104-110. [4] Savage WJ, Brodsky RA. New insights into paroxysmal nocturnal hemoglobinuria[J]. Hematology, 2007, 12(5): 371-376. [5] Hillmen P, Lewis SM, Bessler M, et al. Natural history of paroxysmal nocturnal hemoglobinuria[J]. N Engl J Med, 1995, 333(19):1253-1258. [6] de Latour RP, Mary JY, Salanoubat C, et al. Paroxysmal nocturnal hemoglobinuria: natural history of disease subcategories[J]. Blood, 2008, 112(8):3099-3106. [7] Hill A, Richards SJ, Hillmen P. Recent developments in the understanding and management of paroxysmal nocturnal haemoglobinuria[J]. Br J Haematol, 2007, 137 (3): 181-192. [8] Rother RP, Bell L, Hillmen P, et al. The clinical sequelae of intravascular hemolysis and extracellular plasma hemoglobin[J]. JAMA, 2005, 292 (13): 1653-1662. [9] Hillmen P, Hall C, Marsh JC, et al. Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria[J]. N Engl J Med, 2004, 350(6):552- 559. [10]Brodsky RA, Young NS, Antonioli E, et al. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria[J]. Blood, 2008, 111(4):1840-1847. [11]Hillmen P, Muus P, Dührsen U, et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria[J]. Blood, 2007, 110(12):4123-4128. [12] Höchsmann B, Leichtle R, von Zabern I, et al. Paroxysmal nocturnal hemoglobinuria treatment with eculizumab is associated with a positive direct antiglobulin test[J]. Vox Sang, 2012,102(2):159-166. [13]Risitano AM, Notaro R, Luzzatto L, et al. Paroxysmal nocturnal hemoglobinuria - hemolysis before and after eculizumab[J]. N Engl J Med, 2010, 363(23):2270-2272. [14]Kristiansen M, Graversen JH, Jacobsen C, et al. Identification of the haemoglobin scavenger receptor[J]. Nature, 2001, 409(6817):198-201. [15]Olson JS, Foley EW, Rogge C, et al. NO scavenging and the hypertensive effect of hemoglobin-based blood substitutes[J]. Free Radic Biol Med, 2004, 36(6):685-697. [16]Hill A, Rother RP, Wang X, et al. Effect of eculizumab on haemolysis-associated nitric oxide depletion, dyspnoea, and measures of pulmonary hypertension in patients with paroxysmal nocturnal haemoglobinuria[J]. Br J Haematol, 2010, 149(3):414-425. [17]Schafer A, Wiesmann F, Neubauer S, et al. Rapid regulation of platelet activation in vivo by nitric oxide[J]. Circulation, 2004, 109(15):1819-1822. [18]Matejovic M, Krouzecky A, Radej J, et al. Coagulation and endothelial dysfunction during longterm hyperdynamic porcine bacteremia - effects of selective inducible nitric oxide synthase inhibition[J]. Thromb Haemost, 2007, 97(2):304-309. [19]Gralnick HR, Vail M, McKeown LP, et al. Activated platelets in paroxysmal nocturnal haemoglobinuria[J]. Br J Haematol, 1995, 91(3):697-702. [20]Hugel B, Socie G, Vu T, et al. Elevated levels of circulating procoagulant microparticles in patients with paroxysmal nocturnal hemoglobinuria and aplastic anemia[J]. Blood, 1999, 93(10):3451-3456. [121]Helley D, de Latour RP, Porcher R, et al. Evaluation of hemostasis and endothelial function in patients with paroxysmal nocturnal hemoglobinuria receiving eculizumab[J]. Haematologica, 2010, 95(4):574-581. [22]Kelly RJ, Hill A, Arnold LM, et al. Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival[J]. Blood, 2011, 117(25):6786-6792. [23]Hillmen P, Elebute M, Kelly R, et al. Long-term effect of the complement inhibitor eculizumab on kidney function in patients with paroxysmal nocturnal hemoglobinuria[J]. Am J Hematol, 2010, 85(8):553-559. [24]Nishimura J, Kanakura Y, Ware RE, et al. Clinical course and flow cytometric analysis of paroxysmal nocturnal hemoglobinuria in the United States and Japan[J]. Medicine (Baltimore), 2004, 83(3):193-207. [25]Scheinberg P,Marte M,Nunez 0,et a1.Paroxysmal nocturnal hemoglobinuria clones in severe Aplastic anemia patients treated with horse antithymoeyte globulin plus cyclosporine[J]. Haematologiea,2010,95:1075-1080. [26]Parker CJ.The pathophysiology of paroxysmal nocturnal hemoglobinuria[J].Exp Hematol,2007,35(4):523-533. [27]Brodsky RA, Young NS, Antonioli E, et a1. Muhicenter phase 3 study of the complement inhibitor eculizumab for the treatnlent of patients with paroxysmal nocturnal henmglobinuria[J]. Blood, 2008, 111(1):1840-l847. [128]Rother RP, Rollins SA, Mojcik CF, et al. Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria[J]. Nat Biotechnol, 2007, 25 (11): 1256-1264. [29]Davis J.Eculizumab[J].Am J Health Syst Pharm,2008,65(17):1609-1615. [30]Schrezenmeier H, Höchsmann B. Drugs that inhibit complement[J]. Transfus Apher Sci, 2012, 46 (1):87-92. [31]Noris M, Remuzzi G. Atypical hemolytic-uremic syndrome[J]. N Engl J Med , 2009, 361(17):1676-1687. [32]Kose O, Zimmerhackl LB, Jungraithmayr T, et al. New treatment options for atypical hemolytic uremic syndrome with the complement inhibitor eculizumab[J]. Semin Thromb Hemost, 2010, 36(6):669-672. [33]Lapeyraque AL, Fremeaux-Bacchi V, Robitaille P. Efficacy of eculizumab in a patient with factor-H-associated atypical hemolytic uremic syndrome[J]. Pediatr Nephrol, 2011, 26(4):621-624. [34]Kupferschmidt K. Infectious diseases. As E. coli outbreak recedes, new questions come to the fore[J]. Science, 2011, 333(6038):27. [35]Biglarnia AR, Nilsson B, Nilsson T, et al. Prompt reversal of a severe complement activation by eculizumab in a patient undergoing intentional ABO-incompatible pancreas and kidney transplantation[J]. Transpl Int, 2011, 24(8):e61-e66. [36]Chandran S, Baxter-Lowe L, Olson JL, et al. Eculizumab for the treatment of de novo thrombotic microangiopathy post simultaneous pancreas-kidney transplantation - a case report[J]. Transplant Proc, 2011, 43(5):2097-2101. [37]Bommer M, Höchsmann B, Flegel W, et al. Successful treatment of complement mediated refractory hemolysis associated with cold and warm autoantibodies using eculizumab[J]. Haematologica, 2011, 94(Suppl. 2):241[abs. 0593]. [38]Fitzpatrick AM, Mann CA, Barry S, et al. An open label clinical trial of complement inhibition in multifocal motor neuropathy[J]. J Peripher Nerv Syst, 2011, 16(2):84-91. |