Evaluation of hemostasis and endothelial function in patients with paroxysmal nocturnal hemoglobinuria receiving eculizumab

Haematologica

Volumn 95, Issue 4, 2010, Pages 574-581

  Helley, Dominique ^a,b,c   de Latour, Régis Peffault ^a,d   Porcher, Raphaël ^a,d   Rodrigues, Celso Arrais ^d   Galy Fauroux, Isabelle ^a,b,c   Matheron, Jeanne ^b   Duval, Arnaud ^b,c   Schved, Jean François ^e   Fischer, Anne Marie ^a,b,c   Socié, Gérard ^a,d  

^a UNIVERSITÉ PARIS DESCARTES   (France)

^b HÔPITAL EUROPÉEN GEORGES POMPIDOU   (France)

^c FACULTÉ DES SCIENCES PHARMACEUTIQUES ET BIOLOGIQUES   (France)

^d SAINT LOUIS HOSPITAL   (France)

^e MONTPELLIER UNIVERSITY HOSPITAL   (France)

Author keywords

Eculizumab; Paroxysmal nocturnal hemoglobinuria; Thrombosis

Indexed keywords

ANTIPLASMIN; ANTIVITAMIN K; D DIMER; DANAPAROID; ECULIZUMAB; FONDAPARINUX; INTERCELLULAR ADHESION MOLECULE 1; PLASMIN; PLASMINOGEN ACTIVATOR INHIBITOR 1; PROTHROMBIN; THROMBIN; THROMBOMODULIN; TISSUE FACTOR PATHWAY INHIBITOR; TISSUE PLASMINOGEN ACTIVATOR; VASCULAR CELL ADHESION MOLECULE 1;

ADULT; ARTICLE; CELL ACTIVATION; CLINICAL ARTICLE; CONTROLLED STUDY; DRUG EFFICACY; ENDOTHELIAL DYSFUNCTION; ENDOTHELIAL MICROPARTICLE; ENDOTHELIUM CELL; FIBRINOLYSIS; HEMOSTASIS; HUMAN; PAROXYSMAL NOCTURNAL HEMOGLOBINURIA; PROTEIN ANALYSIS; PROTEIN BLOOD LEVEL; THROMBOSIS; TREATMENT DURATION; VENOUS THROMBOEMBOLISM;

ADULT; ANTIBODIES, MONOCLONAL; BIOLOGICAL MARKERS; COMPLEMENT C5; ENDOTHELIUM, VASCULAR; HEMOGLOBINURIA, PAROXYSMAL; HEMOSTASIS; HUMANS; IMMUNOTHERAPY; MIDDLE AGED; RECEPTORS, CELL SURFACE; THROMBOSIS;

EID: 77950643154     PISSN: 03906078     EISSN: 15928721     Source Type: Journal    
DOI: 10.3324/haematol.2009.016121     Document Type: Article

References (40)

1. Parker CJ. The pathophysiology of paroxysmal nocturnal hemoglobinuria. Exp Hematol. 2007;35(4):523-33.
2. Rother RP, Bell L, Hillmen P, Gladwin MT. The clinical sequelae of intravascular hemolysis and extracellular plasma hemoglobin: a novel mechanism of human dis- ease. Jama. 2005;293(13):1653-62.
3. Hugel B, Socie G, Vu T, Toti F, Gluckman E, Freyssinet JM, et al. Elevated levels of circulating procoagulant microparticles in patients with paroxysmal nocturnal hemoglobinuria and aplastic anemia. Blood. 1999;93(10):3451-6.
4. Ploug M, Plesner T, Ronne E, Ellis V, Hoyer- Hansen G, Hansen NE, et al. The receptor for urokinase-type plasminogen activator is deficient on peripheral blood leukocytes in patients with paroxysmal nocturnal hemoglobinuria. Blood. 1992;79(6):1447-55.
5. Plesner T, Behrendt N, Ploug M. Structure, function and expression on blood and bone marrow cells of the urokinase-type plasminogen activator receptor, uPAR. Stem Cells. 1997;15(6):398-408.
6. Reiter CD, Wang X, Tanus-Santos JE, Hogg N, Cannon RO 3rd, Schechter AN, et al. Cell-free hemoglobin limits nitric oxide bioavailability in sickle-cell disease. Nat Med. 2002;8(12):1383-9.
7. Schafer A, Wiesmann F, Neubauer S, Eigenthaler M, Bauersachs J, Channon KM. Rapid regulation of platelet activation in vivo by nitric oxide. Circulation. 2004; 109(15):1819-22.
8. Hillmen P, Muus P, Duhrsen U, Risitano AM, Schubert J, Luzzatto L, et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007;110(12):4123-8.
9. Ziakas PD, Poulou LS, Rokas GI, Bartzoudis D, Voulgarelis M. Thrombosis in paroxysmal nocturnal hemoglobinuria: sites, risks, outcome. An overview. J Thromb Haemost. 2007;5(3):642-5.
10. Peffault de Latour R, Mary JY, Salanoubat C, Terriou L, Etienne G, Mohty M, et al. Paroxysmal nocturnal hemoglobinuria: nat- ural history of disease subcategories. Blood. 2008;112(8):3099-106.
11. Moyo VM, Mukhina GL, Garrett ES, Brodsky RA. Natural history of paroxysmal nocturnal haemoglobinuria using modern diagnostic assays. Br J Haematol. 2004; 126(1):133-8.
12. Hall C, Richards S, Hillmen P. Primary pro-phylaxis with warfarin prevents thrombo- sis in paroxysmal nocturnal hemoglobinuria (PNH). Blood. 2003;102(10):3587-91.
13. Brodsky RA, Young NS, Antonioli E, Risitano AM, Schrezenmeier H, Schubert J, et al. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood. 2008;111(4): 1840-7.
14. Hillmen P, Hall C, Marsh JC, Elebute M, Bombara MP, Petro BE, et al. Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2004;350(6):552-9.
15. Hillmen P, Young NS, Schubert J, Brodsky RA, Socie G, Muus P, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006;355(12):1233-43.
16. Hillmen P, Elebute M, Kelly R, Urbano- Ispizua A, Rother RP, Fu CL, et al. High Incidence of Progression to Chronic Renal Insufficiency in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). Blood. 2007;110(11):Abstract 3678.
17. Hill A, Rother RP, Wang X, Sapsford RJ, Collinson PO, Gaze DC, et al. Eculizumab reduces pulmonary hypertension through inhibition of hemolysis-associated nitric oxide consumption in patients with parox- ysmal nocturnal hemoglobinuria. Blood. 2008;112(11):Abstract 486.
18. Griscelli-Bennaceur A, Gluckman E, Scrobohaci ML, Jonveaux P, Vu T, Bazarbachi A, et al. Aplastic anemia and paroxysmal nocturnal hemoglobinuria: search for a pathogenetic link. Blood. 1995;85(5):1354-63.
19. Kakkar VV, Hoppenstead DA, Fareed J, Kadziola Z, Scully M, Nakow R, Breddin HK. Randomized trial of different regimens of heparins and in vivo thrombin generation in acute deep vein thrombosis. Blood. 2002;99(6):1965-70.
20. Liebman HA, Feinstein DI. Thrombosis in patients with paroxysmal noctural hemoglobinuria is associated with markedly elevated plasma levels of leukocyte-derived tissue factor. Thromb Res. 2003;111(4- 5):235-8.
21. Grunewald M, Siegemund A, Grunewald A, Schmid A, Koksch M, Schopflin C, et al. Plasmatic coagulation and fibrinolytic system alterations in PNH: relation to clone size. Blood Coagul Fibrinolysis. 2003; 14(7):685-95.
22. Ploug M, Eriksen J, Plesner T, Hansen NE, Dano K. A soluble form of the glycolipid- anchored receptor for urokinase-type plasminogen activator is secreted from peripheral blood leukocytes from patients with paroxysmal nocturnal hemoglobinuria. Eur J Biochem. 1992;208(2):397-404.
23. Ronne E, Pappot H, Grondahl-Hansen J, Hoyer-Hansen G, Plesner T, Hansen NE, et al. The receptor for urokinase plasminogen activator is present in plasma from healthy donors and elevated in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 1995;89(3):576-81.
24. Simak J, Holada K, Risitano AM, Zivny JH, Young NS, Vostal JG. Elevated circulating endothelial membrane microparticles in paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2004;125(6):804-13.
25. Freestone B, Chong AY, Nuttall S, Blann AD, Lip GY. Soluble E-selectin, von Willebrand factor, soluble thrombomod- ulin, and total body nitrate/nitrite product as indices of endothelial damage/dysfunction in paroxysmal, persistent, and permanent atrial fibrillation. Chest. 2007;132(4): 1253-8.
26. Setty BN, Betal SG, Zhang J, Stuart MJ. Heme induces endothelial tissue factor expression: potential role in hemostatic activation in patients with hemolytic anemia. J Thromb Haemost. 2008;6(12):2202- 9.
27. Weitz IC, Ghods M, Rochanda L, Pravazi P, Zwicker J, Furie B, et al. Eculizumab therapy results in rapid and sustained decreases in markers of thrombin generation and inflammation in patients with PNH. Blood. 2008;112(11):Abstract 156.
28. Marin V, Montero-Julian FA, Gres S, Boulay V, Bongrand P, Farnarier C, et al. The IL-6- soluble IL-6Ralpha autocrine loop of endothelial activation as an intermediate between acute and chronic inflammation: an experimental model involving thrombin. J Immunol. 2001;167(6):3435-42.
29. Dahm A, Van Hylckama Vlieg A, Bendz B, Rosendaal F, Bertina RM, Sandset PM. Low levels of tissue factor pathway inhibitor (TFPI) increase the risk of venous thrombosis. Blood. 2003;101(11):4387-92.
30. Sarig G, Blumenfeld Z, Leiba R, Lanir N, Brenner B. Modulation of systemic hemostatic parameters by enoxaparin during gestation in women with thrombophilia and pregnancy loss. Thromb Haemost. 2005; 94(5):980-5.
31. Sloand EM, Pfannes L, Scheinberg P, More K, Wu CO, Horne M, Young NS. 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(12): 1616-24.
32. Ataga KI. Hypercoagulability and thrombotic complications in hemolytic anemias. Haematologica. 2009;94(11):1481-4.
33. Hattori R, Hamilton KK, McEver RP, Sims PJ. Complement proteins C5b-9 induce secretion of high molecular weight multimers of endothelial von Willebrand factor and translocation of granule membrane protein GMP-140 to the cell surface. J Biol Chem. 1989;264(15):9053-60.
34. Hamilton KK, Ji Z, Rollins S, Stewart BH, Sims PJ. Regulatory control of the terminal complement proteins at the surface of human endothelial cells: neutralization of a C5b-9 inhibitor by antibody to CD59. Blood. 1990;76(12):2572-7.
35. Tedesco F, Pausa M, Nardon E, Introna M, Mantovani A, Dobrina A. The cytolytically inactive terminal complement complex activates endothelial cells to express adhesion molecules and tissue factor procoagulant activity. J Exp Med. 1997;185(9):1619- 27.
36. Hamilton KK, Hattori R, Esmon CT, Sims PJ. Complement proteins C5b-9 induce vesiculation of the endothelial plasma membrane and expose catalytic surface for assembly of the prothrombinase enzyme complex. J Biol Chem. 1990;265(7):3809-14.
37. Brunn GJ, Saadi S, Platt JL. Differential regulation of endothelial cell activation by complement and interleukin 1alpha. Circ Res. 2006;98(6):793-800.
38. Zhang J, Piro O, Lu L, Broze GJ Jr. Glycosyl phosphatidylinositol anchorage of tissue factor pathway inhibitor. Circulation. 2003;108(5):623-7.
39. Maroney SA, Cunningham AC, Ferrel J, Hu R, Haberichter S, Mansbach CM, et al. A GPI-anchored co-receptor for tissue factor pathway inhibitor controls its intracellular trafficking and cell surface expression. J Thromb Haemost. 2006;4(5):1114-24.
40. Almeida AM, Murakami Y, Layton DM, Hillmen P, Sellick GS, Maeda Y, et al. Hypomorphic promoter mutation in PIGM causes inherited glycosylphosphatidylinos- itol deficiency. Nat Med. 2006;12(7):846-51.