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1)Takeda J, Miyata T, Kawagoe K, et al. Deficiency of the GPI anchor caused by a somatic mutation of the PIG-A gene in paroxysmal nocturnal hemoglobinuria. Cell. 1993; 73: 703-11
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2)Parker CJ. Paroxysmal nocturnal hemoglobinuria. Curr Opin Hematol. 2012; 19: 141-8
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3)Rooth A, Duhrsen U. Treatment of paroxysmal nocturnal hemoglobinuria in the era of eculizu-mab. Eur J Haematol. 2011; 87: 473-9
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4)大野良之.「特定疾患治療研究事業未対象疾患の疫学像を把握するための調査研究班」平成11年度研究業績集-最終報告書-.平成12年3月発行(2000年)
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5)de Latour RP, Mary JY, Salanoubat C, et al. Paroxysmal nocturnal hemoglobinuria: natural history of disease subcategories. Blood. 2008; 112: 3099-106
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6)Hillmen P, Muus P, Duhrsen U, et al. Effect of the complement inhibitor eculizumab on thrombo-embolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007; 110: 4123-8
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7)厚生労働科学研究費補助金 難治性疾患克服研究事業. 特発性造血障害に関する調査研究班(主任研究者 小峰光博)/PNH の診断基準と診療の参照ガイド作成のためのワーキンググループ. 発作性夜間ヘモグロビン尿症診療の参照ガイド. 2005
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8)Hillmen P, Lewis SM, Bessler M, et al. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med. 1995; 333: 1253-8
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9)Socie G, Mary JY, de Gramont A, et al. Paroxysmal nocturnal haemoglobinuria: long-term follow-up and prognostic factors. French Society of Haematology. Lancet. 1996; 348(9027): 573-7
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10)Hall C, Richards S, Hillmen P. Primary prophylaxis with warfarin prevents thrombosis in paroxysmal nocturnal hemoglobinuria (PNH). Blood. 2003; 102: 3587-91
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11)Nishimura J, Kanakura Y, Ware RE, et al. Clinical course and flow cytometric analysis of paroxysmal nocturnal hemoglobinuria in the United States and Japan. Medicine (Baltimore). 2004; 83: 193-207
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12)Kumar KS, Malet PF, Rutherford C, et al. Paroxysmal nocturnal hemoglobinuria with no evidence of hemolysis presenting as Budd-Chiari syndrome. Dig Dis Sci. 2000; 45: 2238-9
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14)Aharon A, Tamari T, Brenner B, et al. Monocyte-derived microparticles and exosomes induce procoagulant and apoptotic effects on endothelial cells. Thromb Haemost. 2008; 100: 878-85
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15)Simak J, Holada K, Risitano AM, et al. Elevated circulating endothelial membrane microparticles in paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2004; 125: 804-13
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16)Helley D, de Latour RP, Porcher R, et al. Evaluation of hemostasis and endothelial function in patients with paroxysmal nocturnal hemoglobinuria receiving eculizumab. Haematologica. 2010; 95: 574-81
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17)Gao Z, McAlister VC, Williams GM. Repopulation of liver endothelium by bone-marrow-derived cells. Lancet. 2001; 357(9260): 932-3
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18)Zhang J, Piro O, Lu L, et al. Glycosylphospha-tidylinositol anchorage of tissue factor pathway inhibitor. Circulation. 2003; 108: 623-7
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19)Jankowska AM, Szpurka H, Calabro M, et al. Loss of expression of neutrophil proteinase-3: a factor contributing to thrombotic risk in paroxysmal nocturnal hemoglobinuria. Haematologica. 2011; 96: 954-62
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20)Almeida AM, Murakami Y, Layton DM, et al. Hypomorphic promoter mutation in PIGM causes inherited glycosylphosphatidylinositol deficiency. Nat Med. 2006; 12: 846-51
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21)Devine DV, Siegel RS, Rosse WF. Interactions of the platelets in paroxysmal nocturnal hemo-globinuria with complement. Relationship to defects in the regulation of complement and to platelet survival in vivo. J Clin Invest. 1987; 79: 131-7
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23)Wiedmer T, Hall SE, Ortel TL, et al. Complement-induced vesiculation and exposure of membrane prothrombinase sites in platelets of paroxysmal nocturnal hemoglobinuria. Blood. 1993; 82: 1192-6
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24)Hugel B, Socie G, Vu T, et al. Elevated levels of circulating procoagulant microparticles in patients with paroxysmal nocturnal hemo-globinuria and aplastic anemia. Blood. 1999; 93: 3451-6
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25)Ploug M, Plesner T, Rønne E, et al. The receptor for urokinase-type plasminogen activator is deficient on peripheral blood leukocytes in patients with paroxysmal nocturnal hemo-globinuria. Blood. 1992; 79: 1447-55
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26)Sloand EM, Pfannes L, Scheinberg P, et al. Increased soluble urokinase plasminogen activator receptor (suPAR) is associated with thrombosis and inhibition of plasmin generation in paroxysmal nocturnal hemoglobinuria (PNH) patients. Exp Hematol. 2008; 36: 1616-24
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27)Weitz IC, Razavi P, Rochanda L, et al. Eculizumab therapy results in rapid and sustained decreases in markers of thrombin generation and inflammation in patients with PNH independent of its effects on hemolysis and microparticle formation. Thromb Res. 2012 Apr 27. [Epub ahead of print]
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28)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: 6786-92
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29)Luzzatto L, Gianfaldoni G, Notaro R. Manage-ment of paroxysmal nocturnal haemoglobinuria: a personal view. Br J Haematol. 2011; 153: 709-20
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30)Brodsky RA. How I treat paroxysmal nocturnal hemoglobinuria. Blood. 2009; 113: 6522-7
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31)Emadi A, Brodsky RA. Successful discontinuation of anticoagulation following eculizumab adminis-tration in paroxysmal nocturnal hemoglobinuria. Am J Hematol. 2009; 84: 699-701
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