Eculizumab treatment of atypical hemolytic uremic syndrome

Bibliography

• Rother RP, Rollins SA, Mojcik CF, Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Nat Biotechnol 2007;25(11):1256-64
   PubMed Web of Science ®Google Scholar
• Available from: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm272990.htm [Last accessed 6 October 2012]
   Google Scholar
• Gasser C, Gautier E, Steck A, Hemolytic-uremic syndrome: bilateral necrosis of the renal cortex in acute acquired hemolytic anemia. Schweiz Med Wochenschr 1955;85(38-39):905-9
   Google Scholar
• Tsai HM. Advances in the pathogenesis, diagnosis, and treatment of thrombotic thrombocytopenic purpura. J Am Soc Nephrol 2003;14(4):1072-81
   Google Scholar
• Besbas N, Karpman D, Landau D, A classification of hemolytic uremic syndrome and thrombotic thrombocytopenic purpura and related disorders. Kidney Int 2006;70(3):423-31
   PubMed Web of Science ®Google Scholar
• Copelovitch L, Kaplan BS. The thrombotic microangiopathies. Pediatr Nephrol 2008;23(10):1761-7
   PubMed Web of Science ®Google Scholar
• Karmali MA, Petric M, Lim C, The association between idiopathic hemolytic uremic syndrome and infection by verotoxin-producing Escherichia coli. J Infect Dis 1985;151(5):775-82
   Google Scholar
• Copelovitch L, Kaplan BS. Streptococcus pneumoniae–associated hemolytic uremic syndrome: classification and the emergence of serotype 19A. Pediatrics 2010;125(1):e174-82
   Google Scholar
• Barnard PJ, Kibel M. The Haemolytic-Uraemic Syndrome of Infancy and Childhood. A Report of Eleven Cases. Cent Afr J Med 1965;11:31-4
   Google Scholar
• Fitzpatrick MM, Walters MD, Trompeter RS, Atypical (non-diarrhea-associated) hemolytic-uremic syndrome in childhood. J Pediatr 1993;122(4):532-7
   Google Scholar
• Kaplan BS. Hemolytic uremic syndrome with recurrent episodes: an important subset. Clin Nephrol 1977;8(6):495-8
   Google Scholar
• Kaplan BS, Chesney RW, Drummond KN. Hemolytic uremic syndrome in families. N Engl J Med 1975;292(21):1090-3
   Google Scholar
• Loirat C, Fremeaux-Bacchi V. Atypical hemolytic uremic syndrome. Orphanet J Rare Dis 2011;6:60
   PubMed Web of Science ®Google Scholar
• Noris M, Remuzzi G. Atypical hemolytic-uremic syndrome. N Engl J Med 2009;361(17):1676-87
   PubMed Web of Science ®Google Scholar
• Sellier-Leclerc AL, Fremeaux-Bacchi V, Dragon-Durey MA, Differential impact of complement mutations on clinical characteristics in atypical hemolytic uremic syndrome. J Am Soc Nephrol 2007;18(8):2392-400
   PubMed Web of Science ®Google Scholar
• Kaplan BS, Papadimitriou M, Brezin JH, Renal transplantation in adults with autosomal recessive inheritance of hemolytic uremic syndrome. Am J Kidney Dis 1997;30(6):760-5
   Google Scholar
• Kavanagh D, Richards A, Goodship T, Jalanko H. Transplantation in atypical hemolytic uremic syndrome. Semin Thromb Hemost 2010;36(6):653-9
   Google Scholar
• Kavanagh D, Goodship T. Genetics and complement in atypical HUS. Pediatr Nephrol 2010;25(12):2431-42
   Google Scholar
• Noris M, Caprioli J, Bresin E, Relative role of genetic complement abnormalities in sporadic and familial aHUS and their impact on clinical phenotype. Clin J Am Soc Nephrol 2010;5(10):1844-59
   PubMed Web of Science ®Google Scholar
• Walport MJ. Complement. First of two parts. N Engl J Med 2001;344(14):1058-66
   PubMed Web of Science ®Google Scholar
• Zipfel PF, Mache C, Muller D, DEAP-HUS: deficiency of CFHR plasma proteins and autoantibody-positive form of hemolytic uremic syndrome. Pediatr Nephrol 2010;25(10):2009-19
   Google Scholar
• Delvaeye M, Noris M, De Vriese A, Thrombomodulin mutations in atypical hemolytic-uremic syndrome. N Engl J Med 2009;361(4):345-57
   PubMed Web of Science ®Google Scholar
• Tsai HM. Excessive activation of the complement system in atypical hemolytic uremic syndrome: is it ready for prime time? Kidney Int 2010;77(4):267-9
   Google Scholar
• Ruebner RL, Kaplan BS, Copelovitch L. A time for reappraisal of "atypical" hemolytic uremic syndrome: should all patients be treated the same? Eur J Pediatr 2012;171(10):1519-25
   Google Scholar
• Noris M, Bresin E, Mele C, Atypical hemolytic-uremic syndrome. In: Pagon RA, Bird TD, Dolan CR, Stephens K, Adam MP, editors. GeneReviews™ [Internet]. University of Washington, Seattle; Seattle (WA): 1993. 2007 Nov 16 [updated 2011 Mar 10]
   Google Scholar
• Ariceta G, Besbas N, Johnson S, Guideline for the investigation and initial therapy of diarrhea-negative hemolytic uremic syndrome. Pediatr Nephrol 2009;24(4):687-96
   Web of Science ®Google Scholar
• Waters AM, Licht C. aHUS caused by complement dysregulation: new therapies on the horizon. Pediatr Nephrol 2011;26(1):41-57
   Google Scholar
• Roumenina LT, Loirat C, Dragon-Durey MA, Alternative complement pathway assessment in patients with atypical HUS. J Immunol Methods 2011;365(1-2):8-26
   Google Scholar
• Greenbaum L, Babu S, Furman R, Continued improvements in renal function with sustained eculizumab in patients with atypical hemolytic uremic syndrome resistant to plasma exchange/infusion. Abstract presented at the 44th annual meeting of the American Society of Nephrology; 8 – 13 November 2011; Philadelphia, PA, USA
   Google Scholar
• Licht C, Muus P, Legendre C, Phase II study of eculizumab in patients with atypical hemolytic uremic syndrome (aHUS) receiving chronic plasma exchange/infusion. Abstract presented at the 44th annual meeting of the American Society of Nephrology; 8 – 13 November 2011; Philadelphia, PA, USA
   Google Scholar
• Loirat C, Babu S, Furman R, Eculizumab efficacy and safety in patients with atypical hemolytic uremic syndrome (aHUS) resistant to plasma exchange/infusion. Presented at the 16th Congress of European Hematology Association (EHA); London, UK; 2011
   Google Scholar
• Loirat C, Muus P, Legendre C, Phase II study of Eculizumab in patients with atypical hemolytic uremic syndrome receiving chronic plasma exchange/infusion. Presented at the 16th Congress of European Hematology Association (EHA); London, UK; 2011
   Google Scholar
• Muus P, Legendre C, Douglas K, al E. Safety and efficacy of eculizumab in aHUS patients on chronic plasma therapy: interim analysis of a phase 2 trial. Abstract presented at the 43rd annual meeting of the American Society of Nephrology; 16 – 21 November 2010; Denver, CO, USA
   Google Scholar
• Soliris (eculizumab). Prescribing information. Alexion Pharmaceuticals, Inc; Available from: http://www.businesswire.com/news/home/20110923005612/en/Soliris%C2%AE-eculizumab-Approved-FDA-Patients-Atypical-Hemolytic 2011
   Google Scholar
• Simonetti G, Gruppo R, Rodig N, Eculizumab therapy for atypical hemolytic uremic syndrome in pediatric patients: efficacy and safety outcomes from a retrospective study. Presented at the 16th Congress of European Hematology Association (EHA); London, UK; 2011
   Google Scholar
• Kose O, Zimmerhackl LB, Jungraithmayr T, New treatment options for atypical hemolytic uremic syndrome with the complement inhibitor eculizumab. Semin Thromb Hemost 2010;36(6):669-72
   Google Scholar
• Nurnberger J, Philipp T, Witzke O, Eculizumab for atypical hemolytic-uremic syndrome. N Engl J Med 2009;360(5):542-4
   PubMed Web of Science ®Google Scholar
• Gruppo RA, Rother RP. Eculizumab for congenital atypical hemolytic-uremic syndrome. N Engl J Med 2009;360(5):544-6
   PubMed Web of Science ®Google Scholar
• Kim JJ, Waller SC, Reid CJ. Eculizumab in atypical haemolytic uraemic syndrome allows cessation of plasma exchange and dialysis. Clin Kidney J 2012;5:34-6
   Google Scholar
• Lapeyraque AL, Fremeaux-Bacchi V, Robitaille P. Efficacy of eculizumab in a patient with factor-H-associated atypical hemolytic uremic syndrome. Pediatr Nephrol 2011;26(4):621-4
   PubMed Web of Science ®Google Scholar
• Mache CJ, Acham-Roschitz B, Fremeaux-Bacchi V, Complement inhibitor eculizumab in atypical hemolytic uremic syndrome. Clin J Am Soc Nephrol 2009;4(8):1312-16
   Web of Science ®Google Scholar
• Tschumi S, Gugger M, Bucher BS, Eculizumab in atypical hemolytic uremic syndrome: long-term clinical course and histological findings. Pediatr Nephrol 2011;26(11):2085-8
   Google Scholar
• Davin JC, Gracchi V, Bouts A, 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(4):708-11
   Google Scholar
• Prescott HC, Wu HM, Cataland SR, Baiocchi RA. Eculizumab therapy in an adult with plasma exchange-refractory atypical hemolytic uremic syndrome. Am J Hematol 2010;85(12):976-7
   Google Scholar
• Ariceta G, Arrizabalaga B, Aguirre M, Eculizumab in the treatment of atypical hemolytic uremic syndrome in infants. Am J Kidney Dis 2012;59(5):707-10
   PubMed Web of Science ®Google Scholar
• Weitz M, Amon O, Bassler D, Prophylactic eculizumab prior to kidney transplantation for atypical hemolytic uremic syndrome. Pediatr Nephrol 2011;26(8):1325-9
   Google Scholar
• Chandran S, Baxter-Lowe L, Olson JL, Eculizumab for the treatment of de novo thrombotic microangiopathy post simultaneous pancreas-kidney transplantation – a case report. Transplant Proc 2011;43(5):2097-101
   Google Scholar
• Al-Akash SI, Almond PS, Savell VH Jr, Eculizumab induces long-term remission in recurrent post-transplant HUS associated with C3 gene mutation. Pediatr Nephrol 2011;26(4):613-19
   Google Scholar
• Chatelet V, Fremeaux-Bacchi V, Lobbedez T, Safety and long-term efficacy of eculizumab in a renal transplant patient with recurrent atypical hemolytic-uremic syndrome. Am J Transplant 2009;9(11):2644-5
   Google Scholar
• Larrea CF, Cofan F, Oppenheimer F, Efficacy of eculizumab in the treatment of recurrent atypical hemolytic-uremic syndrome after renal transplantation. Transplantation 2010;89(7):903-4
   Google Scholar
• Nester C, Stewart Z, Myers D, Pre-emptive eculizumab and plasmapheresis for renal transplant in atypical hemolytic uremic syndrome. Clin J Am Soc Nephrol 2011;6(6):1488-94
   Google Scholar
• Duran CE, Maduell FM, Campistol JM. Rescue therapy with eculizumab in a transplant recipient with atypical haemolytic uraemic syndrome. Clin Kidney J 2012;5:28-30
   Google Scholar
• Garjau M, Azancot M, Ramos R, Early treatment with eculizumab may be beneficial in atypical haemolytic uraemic syndrome. Clin Kidney J 2012;5:31-3
   PubMedGoogle Scholar
• Krid S, Roumenina LT, Beury D, Renal transplantation under prophylactic eculizumab in atypical hemolytic uremic syndrome with CFH/CFHR1 hybrid protein. Am J Transplant 2012;12(7):1938-44
   Google Scholar
• Zimmerhackl LB, Hofer J, Cortina G, Prophylactic eculizumab after renal transplantation in atypical hemolytic-uremic syndrome. N Engl J Med 2010;362(18):1746-8
   Google Scholar
• Hodgkins KS, Bobrowski AE, Lane JC, Langman CB. Clinical grand rounds: atypical hemolytic uremic syndrome. Am J Nephrol 2012;35(5):394-400
   Google Scholar
• Zuber J, Quintrec ML, Krid S, Eculizumab for atypical hemolytic uremic syndrome recurrence in renal transplantation. Am J Transplant 2012;
   Google Scholar
• 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
   PubMedGoogle Scholar
• Noone D, Al-Matrafi J, Tinckam K, Antibody mediated rejection associated with complement factor h-related protein 3/1 deficiency successfully treated with eculizumab. Am J Transplant 2012;12(9):2546-53
   Google Scholar
• Available from: http://soliris.net/sites/default/files/assets/soliris_pi.pdf [Last accessed 6 October 2012]
   Google Scholar
• Kelly R, Arnold L, Richards S, The management of pregnancy in paroxysmal nocturnal haemoglobinuria on long term eculizumab. Br J Haematol 2010;149(3):446-50
   Google Scholar
• Available from: http://www.alxn.com/SolirisAndPNH/ImportantSafetyInfo.aspx [Last accessed 6October 2012]
   Google Scholar
• Bouts A, Monnens L, Davin JC, Insufficient protection by Neisseria meningitidis vaccination alone during eculizumab therapy. Pediatr Nephrol 2011;26(10):1919-20
   Google Scholar
• Zlamy M, Hofer J, Elias J, Immunogenicity of meningococcus C vaccination in a patient with atypical hemolytic uremic syndrome (aHUS) on eculizumab therapy. Pediatr Transplant 2012;16(6):E246-50
   Google Scholar
• Kim JJ, Goodship TH, Tizard J, Inward C. Plasma therapy for atypical haemolytic uraemic syndrome associated with heterozygous factor H mutations. Pediatr Nephrol 2011;26(11):2073-6
   Google Scholar
• Zuber J, Le Quintrec M, Sberro-Soussan R, New insights into postrenal transplant hemolytic uremic syndrome. Nat Rev Nephrol 2011;7(1):23-35
   PubMed Web of Science ®Google Scholar
• Jalanko H, Peltonen S, Koskinen A, Successful liver-kidney transplantation in two children with aHUS caused by a mutation in complement factor H. Am J Transplant 2008;8(1):216-21
   Google Scholar
• Saland JM, Ruggenenti P, Remuzzi G. Liver-kidney transplantation to cure atypical hemolytic uremic syndrome. J Am Soc Nephrol 2009;20(5):940-9
   Google Scholar
• Zuber J, Fakhouri F, Roumenina LT, Use of eculizumab for atypical haemolytic uraemic syndrome and C3 glomerulopathies. Nat Rev Nephrol 2012 Nov;8(11):643-57
   PubMed Web of Science ®Google Scholar