Anticorps monoclonal anti-C5 (éculizumab) dans l’hémoglobinurie paroxystique nocturne

Gérard Socié; Nathalie Varoqueaux; Régis Peffault de Latour

doi:10.1051/medsci/200925121126

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Volume 25 / Numéro 12 (Décembre 2009)

Med Sci (Paris), 25 12 (2009) 1126-1129

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Numéro		Med Sci (Paris) Volume 25, Numéro 12, Décembre 2009 Anticorps monoclonaux en thérapeutique


Page(s)		1126 - 1129
Section		II - La réalité clinique
DOI		https://doi.org/10.1051/medsci/200925121126
Publié en ligne		15 décembre 2009

Rosse WF, Ware RE. The molecular basis of paroxysmal nocturnal hemoglobinuria. Blood 1995; 86 : 3277–86. [Google Scholar]
Rotoli B, Luzzatto L. Paroxysmal nocturnal haemoglobinuria. Baillieres Clin Haematol 1989; 2 : 113–38. [Google Scholar]
Miyata T, Takeda J, Iida Y, et al. The cloning of PIG-A, a component in the early step of GPI-anchor biosynthesis. Science 1993; 259 : 1318–20. [Google Scholar]
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. [Google Scholar]
Luzzatto L, Bessler M. The dual pathogenesis of paroxysmal nocturnal hemoglobinuria. Curr Opin Hematol 1996; 3 : 101–10. [Google Scholar]
Miyata T, Yamada N, Lida Y, et al. Abnormalities of PIG-A transcripts in granulocytes from patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med 1994; 330 : 249–55. [Google Scholar]
Nafa K, Mason PJ, Hillmen P, et al. Mutations in the PIG-A gene causing paroxysmal nocturnal hemoglobinuria are mainly of the frameshift type. Blood 1995; 86 : 4650–5. [Google Scholar]
Rother RP, Bell L, Hillmen P, Gladwin MT. The clinical sequelae of intravascular hemolysis and extracellular plasma hemoglobin : a novel mechanism of human disease. JAMA 2005; 293 : 1653–62. [Google Scholar]
Hugel B, Socie G, Vu T, et al. Elevated levels of circulating procoagulant microparticles in patients with paroxysmal nocturnal hemoglobinuria and aplastic anemia. Blood 1999; 93 : 3451–6. [Google Scholar]
Doeuvre L, Angles-Cano E. Cell-derived microparticles unveil their fibrinolytic and proteolytic function. Med Sci (Paris) 2009; 25 : 37–44. [Google Scholar]
De Latour RP, Mary JY, Salanoubat C, et al. Paroxysmal nocturnal hemoglobinuria : natural history of disease subcategories. Blood 2008; 112 : 3099–106. [Google Scholar]
Parker C, Omine M, Richards S, et al. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood 2005; 106 : 3699–709. [Google Scholar]
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 : 1256–64. [Google Scholar]
Hillmen P, Hall C, Marsh JC, et al. Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med 2004; 350 : 552–9. [Google Scholar]
Hill A, Hillmen P, Richards SJ, et al. Sustained response and long-term safety of eculizumab in paroxysmal nocturnal hemoglobinuria. Blood 2005; 106 : 2559–65. [Google Scholar]
Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med 2006; 355 : 1233–43. [Google Scholar]
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. Blood 2008; 111 : 1840–7. [Google Scholar]
Hillmen P, Muus P, Duhrsen U, et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood 2007; 110 : 4123–8. [Google Scholar]

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[1] Rosse WF, Ware RE. The molecular basis of paroxysmal nocturnal hemoglobinuria. Blood 1995; 86 : 3277–86. [Google Scholar]

[2] Rotoli B, Luzzatto L. Paroxysmal nocturnal haemoglobinuria. Baillieres Clin Haematol 1989; 2 : 113–38. [Google Scholar]

[3] Miyata T, Takeda J, Iida Y, et al. The cloning of PIG-A, a component in the early step of GPI-anchor biosynthesis. Science 1993; 259 : 1318–20. [Google Scholar]

[4] 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. [Google Scholar]

[5] Luzzatto L, Bessler M. The dual pathogenesis of paroxysmal nocturnal hemoglobinuria. Curr Opin Hematol 1996; 3 : 101–10. [Google Scholar]

[6] Miyata T, Yamada N, Lida Y, et al. Abnormalities of PIG-A transcripts in granulocytes from patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med 1994; 330 : 249–55. [Google Scholar]

[7] Nafa K, Mason PJ, Hillmen P, et al. Mutations in the PIG-A gene causing paroxysmal nocturnal hemoglobinuria are mainly of the frameshift type. Blood 1995; 86 : 4650–5. [Google Scholar]

[8] Rother RP, Bell L, Hillmen P, Gladwin MT. The clinical sequelae of intravascular hemolysis and extracellular plasma hemoglobin : a novel mechanism of human disease. JAMA 2005; 293 : 1653–62. [Google Scholar]

[9] Hugel B, Socie G, Vu T, et al. Elevated levels of circulating procoagulant microparticles in patients with paroxysmal nocturnal hemoglobinuria and aplastic anemia. Blood 1999; 93 : 3451–6. [Google Scholar]

[10] Doeuvre L, Angles-Cano E. Cell-derived microparticles unveil their fibrinolytic and proteolytic function. Med Sci (Paris) 2009; 25 : 37–44. [Google Scholar]

[11] De Latour RP, Mary JY, Salanoubat C, et al. Paroxysmal nocturnal hemoglobinuria : natural history of disease subcategories. Blood 2008; 112 : 3099–106. [Google Scholar]

[12] Parker C, Omine M, Richards S, et al. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood 2005; 106 : 3699–709. [Google Scholar]

[13] 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 : 1256–64. [Google Scholar]

[14] Hillmen P, Hall C, Marsh JC, et al. Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med 2004; 350 : 552–9. [Google Scholar]

[15] Hill A, Hillmen P, Richards SJ, et al. Sustained response and long-term safety of eculizumab in paroxysmal nocturnal hemoglobinuria. Blood 2005; 106 : 2559–65. [Google Scholar]

[16] Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med 2006; 355 : 1233–43. [Google Scholar]

[17] 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. Blood 2008; 111 : 1840–7. [Google Scholar]

[18] Hillmen P, Muus P, Duhrsen U, et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood 2007; 110 : 4123–8. [Google Scholar]