|
1）Krawitz PM, Hochsmann B, Murakami Y, et al. A case of paroxysmal nocturnal hemoglobinuria caused by a germline mutation and a somatic mutation in PIGT. Blood. 2013; 122(7): 1312-5
|
|
|
|
2）Rother RP, Rollins SA, Mojcik CF, et al. Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Nat Biotechnol. 2007; 25(11): 1256-64
|
|
|
|
|
|
3）Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006; 355(12): 1233-43
|
|
|
|
|
|
4）Hillmen P, Muus P, Roth A, et al. Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2013; 162(1): 62-73
|
|
|
|
|
|
5）Kelly RJ, Hill A, Arnold LM, et al. Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood. 2011; 117(25): 6786-92
|
|
|
|
|
|
6）Kanakura Y, Ohyashiki K, Shichishima T, et al. Long-term efficacy and safety of eculizumab in Japanese patients with PNH: AEGIS trial. Int J Hematol. 2013; 98(4): 406-16
|
|
|
|
|
7）Risitano AM, Notaro R, Marando L, et al. Complement fraction 3 binding on erythrocytes as additional mechanism of disease in paroxysmal nocturnal hemoglobinuria patients treated by ecu-lizumab. Blood. 2009; 113(17): 4094-100
|
|
|
|
|
|
8）Risitano AM, Notaro R, Luzzatto L, et al. Paroxysmal nocturnal hemoglobinuria--hemolysis before and after eculizumab. N Engl J Med. 2010; 363(23): 2270-2
|
|
|
|
|
|
9）Tomita A, Shirasugi Y, Ito T, et al. Extravascular hemolytic attack after eculizumab therapy for paroxysmal nocturnal hemoglobinuria. Ann Hematol. 2012; 91(7): 1139-41
|
|
|
|
|
|
10）Risitano AM, Notaro R, Pascariello C, et al. The complement receptor 2/factor H fusion protein TT30 protects paroxysmal nocturnal hemoglobinuria erythrocytes from complement-mediated hemolysis and C3 fragment. Blood. 2012; 119(26): 6307-16
|
|
|
|
|
|
11）Rotoli B, Luzzatto L. Paroxysmal nocturnal hemoglobinuria. Semin Hematol. 1989; 26: 201-7
|
|
|
|
|
|
12）Young NS. The problem of clonality in aplastic anemia: Dr Dameshekʼs riddle, restated. Blood. 1992; 79: 1385-92
|
|
|
|
|
|
13）Kinoshita T, Inoue N. Relationship between aplastic anemia and paroxysmal nocturnal hemoglobinuria. Int J Hematol. 2002; 75(2): 117-22
|
|
|
|
|
|
14）Inoue N, Murakami Y, Kinoshita T. Molecular genetics of paroxysmal nocturnal hemoglobinuria. Int J Hematol. 2003; 77(2): 107-12
|
|
|
|
|
|
15）Inoue N, Izui-Sarumaru T, Murakami Y, et al. Molecular basis of clonal expansion of hematopoiesis in 2 patients with paroxysmal nocturnal hemoglobinuria (PNH). Blood. 2006; 108(13): 4232-6
|
|
|
|
|
|
16）Gargiulo L, Lastraioli S, Cerruti G, et al. Highly homologous T-cell receptor beta sequences support a common target for autoreactive T cells in most patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007; 109(11): 5036-42
|
|
|
|
|
|
17）Gargiulo L, Papaioannou M, Sica M, et al. Glycosylphosphatidylinositol-specific, CD1d-restricted T cells in paroxysmal nocturnal hemoglobinuria. Blood. 2013; 121(14): 2753-61
|
|
|
|
|
|
18）Cavazzana-Calvo M, Payen E, Negre O, et al. Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. Nature. 2010; 467(7313): 318-22
|
|
|
|
|
|
19）Ikeda K, Mason PJ, Bessler M. 3ʼUTR-truncated Hmga2 cDNA causes MPN-like hematopoiesis by conferring a clonal growth advantage at the level of HSC in mice. Blood. 2011; 117(22): 5860-9
|
|
|
|
|
|
20）Murakami Y, Inoue N, Shichishima T, et al. Deregulated expression of HMGA2 is implicated in clonal expansion of PIGA deficient cells in paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2012; 156(3): 383-7
|
|
|
|
|