An update for atypical haemolytic uraemic syndrome: Diagnosis and treatment. A consensus document

Nefrologia

Volumn 35, Issue 5, 2015, Pages 421-447

  Campistol, Josep M ^a   Arias, Manuel ^b   Ariceta, Gema ^c   Blasco, Miguel ^a   Espinosa, Laura ^d   Espinosa, Mario ^e   Grinyó, Josep M ^f   Macía, Manuel ^g   Mendizábal, Santiago ^h   Praga, Manuel ⁱ   Román, Elena ^h   Torra, Roser ^j   Valdés, Francisco ^k   Vilalta, Ramón ^c   Rodríguez de Córdoba, Santiago ^l  

^a HOSPITAL CLÍNIC   (Spain)

^b HOSPITAL UNIVERSITARIO MARQUÉS DE VALDECILLA   (Spain)

^c HOSPITAL MATERNO INFANTIL VALL D HEBRON   (Spain)

^d HOSPITAL UNIVERSITARIO LA PAZ   (Spain)

^e HOSPITAL UNIVERSITARIO REINA SOFÍA   (Spain)

^f HOSPITAL UNIVERSITARI DE BELLVITGE   (Spain)

^g HOSPITAL UNIVERSITARIO NUESTRA SEÑORA DE CANDELARIA   (Spain)

^h HOSPITAL UNIVERSITARIO LA FE   (Spain)

ⁱ HOSPITAL UNIVERSITARIO 12 DE OCTUBRE   (Spain)

^j FUNDACIÓ PUIGVERT   (Spain)

^k Servicio de Nefrología Hospital Universitario   (Spain)

^l CENTRO DE INVESTIGACIONES BIOLÓGICAS   (Spain)

more..

Author keywords

Atypical haemolytic uraemic syndrome; Complement; Eculizumab; Thrombotic microangiopathy

Indexed keywords

COMPLEMENT; COMPLEMENT C5A-INHIBITORS; COMPLEMENT COMPONENT C5; ECULIZUMAB; MONOCLONAL ANTIBODY; SERINE PROTEINASE;

ATYPICAL HEMOLYTIC UREMIC SYNDROME; CLASSIFICATION; COMPLEMENT ACTIVATION; CONSENSUS DEVELOPMENT; DISEASE MANAGEMENT; GENETICS; HUMAN; IMMUNOLOGY; KIDNEY TRANSPLANTATION; METABOLISM; PATHOPHYSIOLOGY; PLASMA EXCHANGE; PRACTICE GUIDELINE; PROGNOSIS; RECURRENT DISEASE; THROMBOTIC MICROANGIOPATHIES; VASCULAR ENDOTHELIUM;

ANTIBODIES, MONOCLONAL, HUMANIZED; ATYPICAL HEMOLYTIC UREMIC SYNDROME; COMPLEMENT ACTIVATION; COMPLEMENT C5; COMPLEMENT SYSTEM PROTEINS; DISEASE MANAGEMENT; ENDOTHELIUM, VASCULAR; HUMANS; KIDNEY TRANSPLANTATION; PLASMA EXCHANGE; PROGNOSIS; RECURRENCE; SERINE ENDOPEPTIDASES; THROMBOTIC MICROANGIOPATHIES;

EID: 84951736132     PISSN: None     EISSN: 20132514     Source Type: Journal    
DOI: 10.1016/j.nefroe.2015.11.006     Document Type: Short Survey

References (149)

1. Loirat C., Fremeaux-Bacchi V. Atypical hemolytic uremic syndrome. Orphanet J Rare Dis 2011, 6:60.
2. Loirat C., Saland J., Bitzan M. Management of hemolytic uremic syndrome. Presse Med 2012, 41:e115-e135.
3. Alexion Pharmaceuticals I. Soliris (eculizumab). Ficha técnica; 2012.
4. Rother R.P., Rollins S.A., Mojcik C.F., Brodsky R.A., Bell L. Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Nat Biotechnol 2007, 25:1256-1264.
5. Legendre C.M., Licht C., Muus P., Greenbaum L.A., Babu S., Bedrosian C., et al. Terminal complement inhibitor eculizumab in atypical hemolytic-uremic syndrome. N Engl J Med 2013, 368:2169-2181.
6. Campistol J.M., Arias M., Ariceta G., Blasco M., Espinosa M., Grinyo J.M., et al. An update for atypical haemolytic uraemic syndrome: diagnosis and treatment. A consensus document. Nefrologia 2013, 33:27-45.
7. Furlan M., Robles N., Lammle B. Partial purification and characterization of a protease from human plasma cleaving von Willebrand factor to fragments produced by in vivo proteolysis. Blood 1996, 87:4223-4234.
8. Pisoni R., Ruggenenti P., Remuzzi G. Drug-induced thrombotic microangiopathy: Incidence, prevention and management. Drug Saf 2001, 24:491-501.
9. Siegler R., Oakes R. Hemolytic uremic syndrome: pathogenesis, treatment, and outcome. Curr Opin Pediatr 2005, 17:200-204.
10. Oakes R.S., Siegler R.L., McReynolds M.A., Pysher T., Pavia A.T. Predictors of fatality in postdiarrheal hemolytic uremic syndrome. Pediatrics 2006, 117:1656-1662.
11. Sellier-Leclerc A.L., Fremeaux-Bacchi V., Dragon-Durey M.A., Macher M.A., Niaudet P., Guest G., et al. Differential impact of complement mutations on clinical characteristics in atypical hemolytic uremic syndrome. J Am Soc Nephrol 2007, 18:2392-2400.
12. Noris M., Caprioli J., Bresin E., Mossali C., Pianetti G., Gamba S., et al. Relative role of genetic complement abnormalities in sporadic and familial aHUS and their impact on clinical phenotype. Clin J Am Soc Nephrol 2010, 5:1844-1859.
13. Sullivan M., Erlic Z., Hoffmann M.M., Arbeiter K., Patzer L., Budde K., et al. Epidemiological approach to identifying genetic predispositions for atypical hemolytic uremic syndrome. Ann Hum Genet 2010, 74:17-26.
14. Westra D., Volokhina E., van der Heijden E., Vos A., Huigen M., Jansen J., et al. Genetic disorders in complement (regulating) genes in patients with atypical haemolytic uraemic syndrome (aHUS). Nephrol Dial Transplant 2010, 25:2195-2202.
15. Esparza-Gordillo J., Goicoechea de Jorge E., Buil A., Carreras Berges L., Lopez-Trascasa M., Sanchez-Corral P., et al. Predisposition to atypical hemolytic uremic syndrome involves the concurrence of different susceptibility alleles in the regulators of complement activation gene cluster in 1q32. Hum Mol Genet 2005, 14:703-712.
16. Dragon-Durey M.A., Blanc C., Marliot F., Loirat C., Blouin J., Sautes-Fridman C., et al. The high frequency of complement factor H related CFHR1 gene deletion is restricted to specific subgroups of patients with atypical haemolytic uraemic syndrome. J Med Genet 2009, 46:447-450.
17. Bienaime F., Dragon-Durey M.A., Regnier C.H., Nilsson S.C., Kwan W.H., Blouin J., et al. Mutations in components of complement influence the outcome of factor I-associated atypical hemolytic uremic syndrome. Kidney Int 2010, 77:339-349.
18. Maga T.K., Nishimura C.J., Weaver A.E., Frees K.L., Smith R.J. Mutations in alternative pathway complement proteins in American patients with atypical hemolytic uremic syndrome. Hum Mutat 2010, 31:E1445-E1460.
19. Dragon-Durey M.A., Sethi S.K., Bagga A., Blanc C., Blouin J., Ranchin B., et al. Clinical features of anti-factor H autoantibody-associated hemolytic uremic syndrome. J Am Soc Nephrol 2010, 21:2180-2187.
20. Caprioli J., Noris M., Brioschi S., Pianetti G., Castelletti F., Bettinaglio P., et al. Genetics of HUS: the impact of MCP, CFH, and IF mutations on clinical presentation, response to treatment, and outcome. Blood 2006, 108:1267-1279.
21. Lemaire M., Fremeaux-Bacchi V., Schaefer F., Choi M., Tang W.H., Le Quintrec M., et al. Recessive mutations in DGKE cause atypical hemolytic-uremic syndrome. Nat Genet 2013, 45:531-536.
22. Bruneau S., Neel M., Roumenina L.T., Frimat M., Laurent L., Fremeaux-Bacchi V., et al. Loss of DGK induces endothelial cell activation and death independently of complement activation. Blood 2015, 125:1038-1046.
23. Ozaltin F., Li B., Rauhauser A., An S.W., Soylemezoglu O., Gonul I.I., et al. DGKE variants cause a glomerular microangiopathy that mimics membranoproliferative GN. J Am Soc Nephrol 2013, 24:377-384.
24. Sharma A.P., Greenberg C.R., Prasad A.N., Prasad C. Hemolytic uremic syndrome (HUS) secondary to cobalamin C (cblC) disorder. Pediatr Nephrol 2007, 22:2097-2103.
25. Waters A.M., Kerecuk L., Luk D., Haq M.R., Fitzpatrick M.M., Gilbert R.D., et al. Hemolytic uremic syndrome associated with invasive pneumococcal disease: the United Kingdom experience. J Pediatr 2007, 151:140-144.
26. Allen U., Licht C. Pandemic H1 N1 influenza A infection and (atypical) HUS-more than just another trigger. Pediatr Nephrol 2011, 26:3-5.
27. Besbas N., Karpman D., Landau D., Loirat C., Proesmans W., Remuzzi G., et al. A classification of hemolytic uremic syndrome and thrombotic thrombocytopenic purpura and related disorders. Kidney Int 2006, 70:423-431.
28. Bitzan M., Schaefer F., Reymond D. Treatment of typical (enteropathic) hemolytic uremic syndrome. Semin Thromb Hemost 2010, 36:594-610.
29. Fakhouri F., Roumenina L., Provot F., Sallee M., Caillard S., Couzi L., et al. Pregnancy-associated hemolytic uremic syndrome revisited in the era of complement gene mutations. J Am Soc Nephrol 2010, 21:859-867.
30. Faguer S., Huart A., Frémeaux-Bacchi V., Ribes D., Chauveau D. Eculizumab and drug-induced haemolytic-uraemic syndrome. Clin Kidney J 2013, 6:484-485.
31. Wilson C.H., Brown A.L., White S.A., Goodship T.H., Sheerin N.S., Manas D.M. Successful treatment of de novo posttransplant thrombotic microangiopathy with eculizumab. Transplantation 2011, 92:e42-e43.
32. Jodele S., Fukuda T., Vinks A., Mizuno K., Laskin B.L., Goebel J., et al. Eculizumab therapy in children with severe hematopoietic stem cell transplantation-associated thrombotic microangiopathy. Biol Blood Marrow Transplant 2014, 20:518-525.
33. Burwick R.M., Feinberg B.B. Eculizumab for the treatment of preeclampsia/HELLP syndrome. Placenta 2013, 34:201-203.
34. Hadaya K., Ferrari-Lacraz S., Fumeaux D., Boehlen F., Toso C., Moll S., et al. Eculizumab in acute recurrence of thrombotic microangiopathy after renal transplantation. Am J Transplant 2011, 11:2523-2527.
35. George J.N., Nester C.M. Syndromes of thrombotic microangiopathy. N Engl J Med 2014, 371:654-666.
36. Constantinescu A.R., Bitzan M., Weiss L.S., Christen E., Kaplan B.S., Cnaan A., et al. Non-enteropathic hemolytic uremic syndrome: causes and short-term course. Am J Kidney Dis 2004, 43:976-982.
37. Neuhaus T.J., Calonder S., Leumann E.P. Heterogeneity of atypical haemolytic uraemic syndromes. Arch Dis Child 1997, 76:518-521.
38. Ohanian M., Cable C., Halka K. Eculizumab safely reverses neurologic impairment and eliminates need for dialysis in severe atypical hemolytic uremic syndrome. Clin Pharmacol 2011, 3:5-12.
39. Sallee M., Daniel L., Piercecchi M.D., Jaubert D., Fremeaux-Bacchi V., Berland Y., et al. Myocardial infarction is a complication of factor H-associated atypical HUS. Nephrol Dial Transplant 2010, 25:2028-2032.
40. Loirat C., Noris M., Fremeaux-Bacchi V. Complement and the atypical hemolytic uremic syndrome in children. Pediatr Nephrol 2008, 23:1957-1972.
41. Kaplan B.S., Garcia C.D., Chesney R.W., Segar W.E., Giugno K., Chem R. Peripheral gangrene complicating idiopathic and recessive hemolytic uremic syndromes. Pediatr Nephrol 2000, 14:985-989.
42. Zuber J., le Quintrec M., Sberro-Soussan R., Loirat C., Fremeaux-Bacchi V., Legendre C. New insights into postrenal transplant hemolytic uremic syndrome. Nat Rev Nephrol 2011, 7:23-35.
43. Ardissino G., Tel F., Testa S., Marzano A.V., Lazzari R., Salardi S., et al. Skin involvement in atypical hemolytic uremic syndrome. Am J Kidney Dis 2014, 63:652-655.
44. Law S.K.A., Reid K.B.M. Complement 1995, IRL Press, Oxford.
45. Caprioli J., Bettinaglio P., Zipfel P.F., Amadei B., Daina E., Gamba S., et al. The molecular basis of familial hemolytic uremic syndrome: mutation analysis of factor H gene reveals a hot spot in Short Consensus Repeat 20. J Am Soc Nephrol 2001, 12:297-307.
46. Fremeaux-Bacchi V., Dragon-Durey M.A., Blouin J., Vigneau C., Kuypers D., Boudailliez B., et al. Complement factor I: a susceptibility gene for atypical haemolytic uraemic syndrome. J Med Genet 2004, 41:e84.
47. Fremeaux-Bacchi V., Moulton E.A., Kavanagh D., Dragon-Durey M.-A., Blouin J., Caudy A., et al. Genetic and functional analyses of membrane cofactor protein (CD46) mutations in atypical hemolytic uremic syndrome. J Am Soc Nephrol 2006, 17:2017-2025.
48. Fremeaux-Bacchi V., Miller E.C., Liszewski M.K., Strain L., Blouin J., Brown A.L., et al. Mutations in complement C3 predispose to development of atypical hemolytic uremic syndrome. Blood 2008, 112:4948-4952.
49. Goicoechea de Jorge E., Harris C.L., Esparza-Gordillo J., Carreras L., Arranz E.A., Garrido C.A., et al. Gain-of-function mutations in complement factor B are associated with atypical hemolytic uremic syndrome. Proc Natl Acad Sci U S A 2007, 104:240-245.
50. Kavanagh D., Kemp E.J., Mayland E., Winney R.J., Duffield J.S., Warwick G., et al. Mutations in complement factor I predispose to development of atypical hemolytic uremic syndrome. J Am Soc Nephrol 2005, 16:2150-2155.
51. Noris M., Brioschi S., Caprioli J., Todeschini M., Bresin E., Porrati F., et al. Familial haemolytic uraemic syndrome and an MCP mutation. Lancet 2003, 362:1542-1547.
52. Pérez-Caballero D., González-Rubio C., Gallardo M.E., Vera M., López-Trascasa M., Rodríguez de Córdoba S., et al. Clustering of missense mutations in the C-terminal region of factor h in atypical hemolytic uremic syndrome. Am J Hum Genet 2001, 68:478-484.
53. Richards A., Buddles M.R., Donne R.L., Kaplan B.S., Kirk E., Venning M.C., et al. Factor H mutations in hemolytic uremic syndrome cluster in exons 18-20, a domain important for host cell recognition. Am J Hum Genet 2001, 68:485-490.
54. Richards A., Kemp E.J., Liszewski M.K., Goodship J.A., Lampe A.K., Decorte R., et al. Mutations in human complement regulator, membrane cofactor protein (CD46), predispose to development of familial hemolytic uremic syndrome. Proc Natl Acad Sci U S A 2003, 100:12966-12971.
55. De Cordoba S.R., de Jorge E.G. Translational mini-review series on complement factor H: Genetics and disease associations of human complement factor H. Clin Exp Immunol 2008, 151:1-13.
56. Dragon-Durey M.-A., Loirat C., Cloarec S., Macher M.-A., Blouin J., Nivet H., et al. Anti-factor H autoantibodies associated with atypical hemolytic uremic syndrome. J Am Soc Nephrol 2005, 16:555-563.
57. Jozsi M., Strobel S., Dahse H.-M., Liu W.-S., Hoyer P.F., Oppermann M., et al. Anti factor H autoantibodies block C-terminal recognition function of factor H in hemolytic uremic syndrome. Blood 2007, 110:1516-1518.
58. Abarrategui-Garrido C., Martinez-Barricarte R., Lopez-Trascasa M., de Cordoba S.R., Sanchez-Corral P. Characterization of complement factor H-related (CFHR) proteins in plasma reveals novel genetic variations of CFHR1 associated with atypical hemolytic uremic syndrome. Blood 2009, 114:4261-4271.
59. Caprioli J., Castelletti F., Bucchioni S., Bettinaglio P., Bresin E., Pianetti G., et al. Complement factor H mutations and gene polymorphisms in haemolytic uraemic syndrome: the C-257T, the A2089G and the G2881T polymorphisms are strongly associated with the disease. Hum Mol Genet 2003, 12:3385-3395.
60. Esparza-Gordillo J., Jorge E.G., Garrido C.A., Carreras L., López-Trascasa M., Sánchez-Corral P., et al. Insights into hemolytic uremic syndrome: segregation of three independent predisposition factors in a large, multiple affected pedigree. Mol Immunol 2006, 43:1769-1775.
61. Bresin E., Rurali E., Caprioli J., Sanchez-Corral P., Fremeaux-Bacchi V., Rodriguez de Cordoba S., et al. Combined complement gene mutations in atypical hemolytic uremic syndrome influence clinical phenotype. J Am Soc Nephrol 2013, 24:475-486.
62. Delvaeye M., Noris M., de Vriese A., Esmon C.T., Esmon N.L., del-Favero J., et al. Thrombomodulin mutations in atypical hemolytic-uremic syndrome. N Engl J Med 2009, 361:345-357.
63. Bu F., Maga T., Meyer N., Wang K., Thomas C., Nester C., et al. Comprehensive genetic analysis of complement and coagulation genes in atypical hemolytic uremic syndrome. J Am Soc Nephrol 2014, 25:55-64.
64. Sanchez-Chinchilla D., Pinto S., Hoppe B., Adragna M., Lopez L., Justa Roldan M., et al. Complement mutations in diacylglycerol kinase-e open-associated atypical hemolytic uremic syndrome. Clin J Am Soc Nephrol 2014, 9:1611-1619.
65. Bresin E., Daina E., Noris M., Castelletti F., Stefanov R., Hill P., et al. Outcome of renal transplantation in patients with non-Shiga toxin-associated hemolytic uremic syndrome: Prognostic significance of genetic background. Clin J Am Soc Nephrol 2006, 1:88-99.
66. Loirat C., Fremeaux-Bacchi V. Hemolytic uremic syndrome recurrence after renal transplantation. Pediatr Transplant 2008, 12:619-629.
67. Le Quintrec M., Zuber J., Moulin B., Kamar N., Jablonski M., Lionet A., et al. Complement genes strongly predict recurrence and graft outcome in adult renal transplant recipients with atypical hemolytic and uremic syndrome. Am J Transplant 2013, 13:663-675.
68. Zuber J., Le Quintrec M., Morris H., Fremeaux-Bacchi V., Loirat C., Legendre C. Targeted strategies in the prevention and management of atypical HUS recurrence after kidney transplantation. Transplant Rev (Orlando) 2013, 27:117-125.
69. Al-Akash S.I., Almond P.S., Savell V.H., Gharaybeh S.I., Hogue C. Eculizumab induces long-term remission in recurrent post-transplant HUS associated with C3 gene mutation. Pediatr Nephrol 2011, 26:613-619.
70. Saland J.M., Ruggenenti P., Remuzzi G. Liver-kidney transplantation to cure atypical hemolytic uremic syndrome. J Am Soc Nephrol 2009, 20:940-949. and the Consensus Study G.
71. Venables J.P., Strain L., Routledge D., Bourn D., Powell H.M., Warwicker P., et al. Atypical haemolytic uraemic syndrome associated with a hybrid complement gene. PLoS Med 2006, 3:e431.
72. Roumenina L.T., Jablonski M., Hue C., Blouin J., Dimitrov J.D., Dragon-Durey M.A., et al. Hyperfunctional C3 convertase leads to complement deposition on endothelial cells and contributes to atypical hemolytic uremic syndrome. Blood 2009, 114:2837-2845.
73. Le Quintrec M., Zuber J., Noel L.H., Thervet E., Fremeaux-Bacchi V., Niaudet P., et al. Anti-factor H autoantibodies in a fifth renal transplant recipient with atypical hemolytic and uremic syndrome. Am J Transplant 2009, 9:1223-1229.
74. De Serres S.A., Isenring P. Athrombocytopenic thrombotic microangiopathy, a condition that could be overlooked based on current diagnostic criteria. Nephrol Dial Transplant 2009, 24:1048-1050.
75. Burns E.R., Lou Y., Pathak A. Morphologic diagnosis of thrombotic thrombocytopenic purpura. Am J Hematol 2004, 75:18-21.
76. Levi M. Disseminated intravascular coagulation: what's new?. Crit Care Clin 2005, 21:449-467.
77. Mannucci P.M. Thrombotic thrombocytopenic purpura and the hemolytic uremic syndrome: much progress and many remaining issues. Haematologica 2007, 92:878-880.
78. Alvarez-Larran A., del Rio-Garma J., Pujol M., de la Rubia J., Hernandez-Jodra M., Borrell M., et al. Newly diagnosed versus relapsed idiopathic thrombotic thrombocytopenic purpura: a comparison of presenting clinical characteristics and response to treatment. Ann Hematol 2009, 88:973-978.
79. Tsai H.M. Pathophysiology of thrombotic thrombocytopenic purpura. Int J Hematol 2010, 91:1-19.
80. Contreras E., de la Rubia J., del Rio-Garma J., Díaz-Ricart M., García-Gala J.M., Lozano M. Diagnostic and therapeutic guidelines of thrombotic microangiopathies of the Spanish Apheresis Group. Med Clin (Barc) 2015, 144. por el Grupo Español de Aféresis, 331.e1-331.e13.
81. Loirat C., Garnier A., Sellier-Leclerc A.L., Kwon T. Plasmatherapy in atypical hemolytic uremic syndrome. Semin Thromb Hemost 2010, 36:673-681.
82. Ariceta G., Besbas N., Johnson S., Karpman D., Landau D., Licht C., et al. Guideline for the investigation and initial therapy of diarrhea-negative hemolytic uremic syndrome. Pediatr Nephrol 2009, 24:687-696.
83. Boyer O., Balzamo E., Charbit M., Biebuyck-Gouge N., Salomon R., Dragon-Durey M.A., et al. Pulse cyclophosphamide therapy and clinical remission in atypical hemolytic uremic syndrome with anti-complement factor H autoantibodies. Am J Kidney Dis 2010, 55:923-927.
84. Lionet A., Provôt F., Glowacki F., Frémeaux-Bacchi V., Hazzan M. A case of adult atypical haemolytic uraemic syndrome related to anti-factor H autoantibodies successfully treated by plasma exchange, corticosteroids and rituximab. NDT Plus 2009, 2:458-460.
85. Michon B., Moghrabi A., Winikoff R., Barrette S., Bernstein M.L., Champagne J., et al. Complications of apheresis in children. Transfusion (Paris) 2007, 47:1837-1842.
86. Johnson S., Stojanovic J., Ariceta G., Bitzan M., Besbas N., Frieling M., et al. An audit analysis of a guideline for the investigation and initial therapy of diarrhea negative (atypical) hemolytic uremic syndrome. Pediatr Nephrol 2014, 29:1967-1978.
87. Delmas Y., Loirat C., Muus P., Legendre C., Douglas K., Hourmant M., et al. Eculizumab (ECU) in atypical Hemolytic Uremic Syndrome (aHUS) Patients (Pts) with long disease duration and Chronic Kidney Disease (CKD): Sustained efficacy at 3 years. J Am Soc Nephrol 2013, 24:822A.
88. Gaber A.O., Loirat C., Greenbaum L.A., Babu S., Furman R., Sheerin N., et al. Eculizumab (ECU) maintains efficacy in atypical Hemolytic Uremic Syndrome (aHUS) Patients (Pts) with progressing Thrombotic Microangiopathy (TMA): 3-year (Yr) update. J Am Soc Nephrol 2013, 24:822A.
89. Kelly R.J., Hochsmann B., Szer J., Kulasekararaj A., de Guibert S., Roth A., et al. Eculizumab in pregnant patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med 2015, 373:1032-1039.
90. Fakhouri F., Hourmant M., Campistol J.M., Cataland S.R., Espinosa M., Gaber A.O., et al. Eculizumab (ECU) inhibits Thrombotic Microangiopathy (TMA) and improves renal function in adult atypical Hemolytic Uremic Syndrome (aHUS) patients (Pts). J Am Soc Nephrol 2013, 24:49-50A.
91. Greenbaum L.A., Fila M., Tsimaratos M., Ardissino G., Al-Akash S.I., Evans J., et al. Eculizumab (ECU) inhibits Thrombotic Microangiopathy (TMA) and improves renal function in pediatric atypical Hemolytic Uremic Syndrome (aHUS) patients (Pts). J Am Soc Nephrol 2013, 24:821A.
92. Faas S., Cofiell R., Kukreja A., Bedard K., Yan Y., Mickle A., et al. Reduction of biomarkers related to thrombotic microangiopathy in patients with aHUS treated with eculizumab. Haematologica 2014, 99:473.
93. Zuber J., Fakhouri F., Roumenina L.T., Loirat C., Fremeaux-Bacchi V. Use of eculizumab for atypical haemolytic uraemic syndrome and C3 glomerulopathies. Nat Rev Nephrol 2012, 8:643-657.
94. Fakhouri F., Fremeaux-Bacchi V. Thrombotic microangiopathy: eculizumab for atypical haemolytic uraemic syndrome: what next?. Nat Rev Nephrol 2013, 9:495-496.
95. Fakhouri F., Delmas Y., Provot F., Barbet C., Karras A., Makdassi R., et al. Insights from the use in clinical practice of eculizumab in adult patients with atypical hemolytic uremic syndrome affecting the native kidneys: an analysis of 19 cases. Am J Kidney Dis 2014, 63:40-48.
96. Ardissino G., Testa S., Possenti I., Tel F., Paglialonga F., Salardi S., et al. Discontinuation of eculizumab maintenance treatment for atypical hemolytic uremic syndrome: a report of 10 cases. Am J Kidney Dis 2014, 64:633-637.
97. Legault D.J., Boelkins M.R. Successful treatment of aHUS recurrence and arrest of plasma exchange resistant TMA post-renal transplantation with the terminal complement inhibitor eculizumab. Blood 2009, 114. Abstr 2421.
98. Davin J.C., Gracchi V., Bouts A., Groothoff J., Strain L., Goodship T. Maintenance of kidney function following treatment with eculizumab and discontinuation of plasma exchange after a third kidney transplant for atypical hemolytic uremic syndrome associated with a CFH mutation. Am J Kidney Dis 2010, 55:708-711.
99. Durán C.E., Blasco M., Maduell F., Campistol J.M. Rescue therapy with eculizumab in a transplant recipient with atypical haemolytic uremic syndrome. Clin Kidney J 2012, 5:28-30.
100. Zuber J., Quintrec M.L., Krid S., Bertoye C., Gueutin V., Lahoche A., et al. Eculizumab for atypical hemolytic uremic syndrome recurrence in renal transplantation. Am J Transplant 2012, 12:3337-3354.
101. Jalanko H., Peltonen S., Koskinen A., Puntila J., Isoniemi H., Holmberg C., et al. Successful liver-kidney transplantation in two children with aHUS caused by a mutation in complement factor H. Am J Transplant 2008, 8:216-221.
102. Saland J.M., Shneider B.L., Bromberg J.S., Shi P.A., Ward S.C., Magid M.S., et al. Successful split liver-kidney transplant for factor H associated hemolytic uremic syndrome. Clin J Am Soc Nephrol 2009, 4:201-206.
103. Haller W., Milford D.V., Goodship T.H., Sharif K., Mirza D.F., McKiernan P.J. Successful isolated liver transplantation in a child with atypical hemolytic uremic syndrome and a mutation in complement factor H. Am J Transplant 2010, 10:2142-2147.
104. Wilson C., Torpey N., Jaques B., Strain L., Talbot D., Manas D., et al. Successful simultaneous liver-kidney transplant in an adult with atypical hemolytic uremic syndrome associated with a mutation in complement factor H. Am J Kidney Dis 2011, 58:109-112.
105. Tran H., Chaudhuri A., Concepcion W., Grimm P.C. Use of eculizumab and plasma exchange in successful combined liver-kidney transplantation in a case of atypical HUS associated with complement factor H mutation. Pediatr Nephrol 2014, 29:477-480.
106. Davin J.C., Buter N., Groothoff J., van Wijk J., Bouts A., Strain L., et al. Prophylactic plasma exchange in CD46-associated atypical haemolytic uremic syndrome. Pediatr Nephrol 2009, 24:1757-1760.
107. Zimmerhackl L.B., Hofer J., Cortina G., Mark W., Wurzner R., Jungraithmayr T.C., et al. Prophylactic eculizumab after renal transplantation in atypical hemolytic-uremic syndrome. N Engl J Med 2010, 362:1746-1748.
108. Weitz M., Amon O., Bassler D., Koenigsrainer A., Nadalin S. Prophylactic eculizumab prior to kidney transplantation for atypical hemolytic uremic syndrome. Pediatr Nephrol 2011, 26:1325-1329.
109. Nester C., Stewart Z., Myers D., Jetton J., Nair R., Reed A., et al. Pre-emptive eculizumab and plasmapheresis for renal transplant in atypical hemolytic uremic syndrome. Clin J Am Soc Nephrol 2011, 6:1488-1494.
110. Krid S., Roumenina L., Beury D., Charbit M., Boyer O., Fremeaux-Bacchi V., et al. Renal transplantation under prophylactic eculizumab in atypical hemolytic uremic syndrome with CFH/CFHR1 hybrid protein. Am J Transplant 2012, 12:1938-1944.
111. Roman-Ortiz E., Mendizabal Oteiza S., Pinto S., Lopez-Trascasa M., Sanchez-Corral P., Rodriguez de Cordoba S. Eculizumab long-term therapy for pediatric renal transplant in aHUS with CFH/CFHR1 hybrid gene. Pediatr Nephrol 2014, 29:149-153.
112. Blasco Pelicano M., Rodríguez de Córdoba S., Diekmann F., Saiz M., Herrero S., Oppenheimer F., et al. Anti-C5 as prophylactic therapy in atypical hemolytic uremic syndrome in living-related kidney transplantation. Transplantation 2013, 96:e26-e29.
113. Donne R.L., Abbs I., Barany P., Elinder C.-G., Little M., Conlon P., et al. Recurrence of hemolytic uremic syndrome after live related renal transplantation associated with subsequent de novo disease in the donor. Am J Kidney Dis 2002, 40:E22.
114. Naesens M., Kuypers D.R., Sarwal M. Calcineurin inhibitor nephrotoxicity. Clin J Am Soc Nephrol 2009, 4:481-508.
115. Reynolds J.C., Agodoa L.Y., Yuan C.M., Abbott K.C. Thrombotic microangiopathy after renal transplantation in the United States. Am J Kidney Dis 2003, 42:1058-1068.
116. Robson M., Cote I., Abbs I., Koffman G., Goldsmith D. Thrombotic micro-angiopathy with sirolimus-based immunosuppression: potentiation of calcineurin-inhibitor-induced endothelial damage. Am J Transplant 2003, 3:324-327.
117. Murphy T., Maw D., Besser M., Sureda S. The successful treatment of transplant-associated thrombotic microangiopathy with eculuzimab P632. Bone Marrow Transplant 2012, 47:S1-S527.
118. Chandran S., Baxter-Lowe L., Olson J.L., Tomlanovich S.J., Webber A. Eculizumab for the treatment of de novo thrombotic microangiopathy post simultaneous pancreas-kidney transplantation-a case report. Transplant Proc 2011, 43:2097-2101.
119. Safa K., Logan M.S., Batal I., Gabardi S., Rennke H.G., Abdi R. Eculizumab for drug-induced de novo post-transplantation thrombotic microangiopathy: a case report. Clin Nephrol 2015, 83:125-129.
120. Peffault de Latour R., Xhaard A., Fremeaux-Bacchi V., Coppo P., Fischer A.M., Helley D., et al. Successful use of eculizumab in a patient with post-transplant thrombotic microangiopathy. Br J Haematol 2013, 161:279-280.
121. Gruppo R.A., Rother R.P. Eculizumab for congenital atypical hemolytic-uremic syndrome. N Engl J Med 2009, 360:544-546.
122. Gruppo R.A., Dixon B.P. Long-term outcome in a pediatric patient with atypical hemolytic uremic syndrome (aHUS) with sustained eculizumab (ECU) treatment. Blood 2011, 118. Abstr 4682.
123. Fremont O.T., Gordon C.A., Hand M.M. Eculizumab treatment for aHUS in a child with positive family history. J Am Soc Nephrol 2009, 20:988 A. Abstr PUB715.
124. Mache C.J., Acham-Roschitz B., Fremeaux-Bacchi V., Kirschfink M., Zipfel P.F., Roedl S., et al. Complement inhibitor eculizumab in atypical hemolytic uremic syndrome. Clin J Am Soc Nephrol 2009, 4:1312-1316.
125. Kose O., Zimmerhackl L.B., Jungraithmayr T., Mache C., Nurnberger J. New treatment options for atypical hemolytic uremic syndrome with the complement inhibitor eculizumab. Semin Thromb Hemost 2010, 36:669-672.
126. Lapeyraque A.L., Fremeaux-Bacchi V., Robitaille P. Efficacy of eculizumab in a patient with factor-H-associated atypical hemolytic uremic syndrome. Pediatr Nephrol 2011, 26:621-624.
127. Prescott H.C., Wu H.M., Cataland S.R., Baiocchi R.A. Eculizumab therapy in an adult with plasma exchange-refractory atypical hemolytic uremic syndrome. Am J Hematol 2010, 85:976-977.
128. Ohanian M., Cable C., Halka K. Reduced dose maintenance eculizumab in atypical hemolytic uremic syndrome (aHUS): an update on a previous case report. Clin Pharmacol 2011, 3:45-50.
129. Tschumi S., Gugger M., Bucher B.S., Riedl M., Simonetti G.D. Eculizumab in atypical hemolytic uremic syndrome: long-term clinical course and histological findings. Pediatr Nephrol 2011, 26:2085-2088.
130. Dorresteijn E.M., van de Kar N.C., Cransberg K. Eculizumab as rescue therapy for atypical hemolytic uremic syndrome with normal platelet count. Pediatr Nephrol 2012, 27:1193-1195.
131. Kim J.J., Waller S.C., Reid C.J. Eculizumab in atypical haemolytic-uraemic syndrome allows cessation of plasma exchange and dialysis. Clin Kidney J 2012, 5:34-36.
132. Garjau M., Azancot M., Ramos R., Sánchez-Corral P., Montero M.A., Serón D. Early treatment with eculizumab in atypical haemolytic uraemic syndrome. Clin Kidney J 2012, 5:31-33.
133. Ariceta G., Arrizabalaga B., Aguirre M., Morteruel E., Lopez-Trascasa M. Eculizumab in the treatment of atypical hemolytic uremic syndrome in infants. Am J Kidney Dis 2012, 59:707-710.
134. Vilalta R., Lara E., Madrid A., Chocron S., Munoz M., Casquero A., et al. Long-term eculizumab improves clinical outcomes in atypical hemolytic uremic syndrome. Pediatr Nephrol 2012, 27:2323-2326.
135. Carr R., Cataland S.R. Relapse of aHUS after discontinuation of therapy with eculizumab in a patient with aHUS and factor H mutation. Ann Hematol 2013, 92:845-846.
136. Thajudeen B., Sussman A., Bracamonte E. A case of atypical hemolytic uremic syndrome successfully treated with eculizumab. Case Rep Nephrol Urol 2013, 3:139-146.
137. Malina M., Gulati A., Bagga A., Majid M.A., Simkova E., Schaefer F. Peripheral gangrene in children with atypical hemolytic uremic syndrome. Pediatrics 2013, 131:e331-e335.
138. Giordano M., Castellano G., Messina G., Divella C., Bellantuono R., Puteo F., et al. Preservation of renal function in atypical hemolytic uremic syndrome by eculizumab: a case report. Pediatrics 2012, 130:e1385-e1388.
139. Povey H., Vundru R., Junglee N., Jibani M. Renal recovery with eculizumab in atypical hemolytic uremic syndrome following prolonged dialysis. Clin Nephrol 2013, 82:326-331.
140. Salem G., Flynn J.M., Cataland S.R. Profound neurological injury in a patient with atypical hemolytic uremic syndrome. Ann Hematol 2013, 92:557-558.
141. Gilbert R.D., Fowler D.J., Angus E., Hardy S.A., Stanley L., Goodship T.H. Eculizumab therapy for atypical haemolytic uraemic syndrome due to a gain-of-function mutation of complement factor B. Pediatr Nephrol 2013, 28:1315-1318.
142. Ardissino G., Wally Ossola M., Baffero G.M., Rigotti A., Cugno M. Eculizumab for atypical hemolytic uremic syndrome in pregnancy. Obstet Gynecol 2013, 122:487-489.
143. Delmas Y., Bordes C., Loirat C., Fremeaux-Bacchi V., Combe C. Post-partum atypical haemolytic-uraemic syndrome treated with eculizumab: terminal complement activity assessment in clinical practice. Clin Kidney J 2013, 6:234-244.
144. Zschiedrich S., Prager E.P., Kuehn E.W. Successful treatment of the postpartum atypical hemolytic uremic syndrome with eculizumab. Ann Intern Med 2013, 159:76.
145. Nürnberger J., Philipp T., Witzke O., Saez A.O., Vester U., Baba H.A., et al. Eculizumab for atypical hemolytic uremic syndrome. N Engl J Med 2009, 360:542-544.
146. Chatelet V., Lobbedez T., Fremeaux-Bacchi V., Ficheux M., Ryckelynck J.P., Hurault de Ligny B. Eculizumab: safety and efficacy after 17 months of treatment in a renal transplant patient with recurrent atypical hemolytic-uremic syndrome: Case report. Transplant Proc 2010, 42:4353-4355.
147. Chatelet V., Fremeaux-Bacchi V., Lobbedez T., Ficheux M., Hurault de Ligny B. Safety and long-term efficacy of eculizumab in a renal transplant patient with recurrent atypical hemolytic-uremic syndrome. Am J Transplant 2009, 9:2644-2645.
148. Larrea C.F., Cofan F., Oppenheimer F., Campistol J.M., Escolar G., Lozano M. Efficacy of eculizumab in the treatment of recurrent atypical hemolytic-uremic syndrome after renal transplantation. Transplantation 2010, 89:903-904.
149. Alachkar N., Bagnasco S.M., Montgomery R.A. Eculizumab for the treatment of two recurrences of atypical hemolytic uremic syndrome in a kidney allograft. Transpl Int 2012, 25:e93-e95.