74
Views
6
CrossRef citations to date
0
Altmetric
Review

Critical appraisal of eculizumab for atypical hemolytic uremic syndrome

&
Pages 39-72 | Published online: 12 Apr 2016

References

  • NorisMRemuzziGGlomerular diseases dependent on complement activation, including atypical hemolytic uremic syndrome, membranoproliferative glomerulonephritis, and C3 glomerulopathy: core curriculum 2015Am J Kidney Dis201566235937526032627
  • GilbertRDStanleyLKFowlerDJAngusEMHardySAGoodshipTHCisplatin-induced haemolytic uraemic syndrome associated with a novel intronic mutation of treated with eculizumabClin Kidney J20136442142524422172
  • FaguerSHuartAFremeaux-BacchiVRibesDChaveauDEculizumab and drug-induced haemolytic-uraemic syndromeClin Kidney J20136548448526120441
  • DahabrehITsoutsosGTseligasDJaninisDHemolytic uremic syndrome following the infusion of oxaliplatin: case reportBMC Clin Pharmacol20066516968538
  • BuFBorsaNGJonesMBHigh-throughput genetic testing for thrombotic microangiopathies and C3 glomerulopathiesJ Am Soc Nephrol Epub2015817
  • JodeleSZhangKZouFThe genetic fingerprint of susceptibility for transplant-associated thrombotic microangiopathyBlood2016127898999626603840
  • ThomasTCRollinsSARotherRPInhibition of complement activity by humanized anti-C5 antibody and single-chain FvMol Immunol19963317–18138914019171898
  • RotherRPRollinsSAMojcikCFBrodskyRABellLDiscovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuriaNat Biotechnol200725111256126417989688
  • US Food and Drug AdministrationSoliris® (eculizumab) [prescribing information]Cheshire, CTAlexion Pharmaceuticals, Inc2014
  • European Medicines Agency Soliris® (eculizumab) [summary of product characteristics]Paris, FranceAlexion Europe SAS2015
  • LoiratCFakhouriFAricetaGAn international consensus approach to the management of atypical hemolytic uremic syndrome in childrenPediatr Nephrol2016311153925859752
  • Peffault de LatourRFremeaux-BacchiVPorcherRAssessing complement blockade in patients with paroxysmal nocturnal hemoglobinuria receiving eculizumabBlood2015125577578325477495
  • SheerinNSKavanaghDGoodshipTHJohnsonSA national specialized service in England for atypical haemolytic uraemic syndrome-the first year’s experienceQJM20161091273325899302
  • JodeleSFukudaTVinksAEculizumab therapy in children with severe hematopoietic stem cell transplantation-associated thrombotic microangiopathyBiol Blood Marrow Transplant201420451852524370861
  • NorisMGalbuseraMGastoldiSDynamics of complement activation in atypical HUS and how to monitor eculizumab therapyBlood2014124111715172625037630
  • BellWRBraineHGNessPMKicklerTSImproved survival in thrombotic thrombocytopenic purpura-hemolytic uremic syndrome. Clinical experience in 108 patientsN Engl J Med199132563984032062331
  • SchieppatiARuggenentiPCornejoRPRenal function at hospital admission as a prognostic factor in adult hemolytic uremic syndrome. The Italian Registry of Haemolytic Uremic SyndromeJ Am Soc Nephrol1992211164016441610985
  • TaylorCMChuaCHowieAJRisdonRABritish Association for Paediatric NephrologyClinicopathological findings in diarrhoea-negative haemolytic uraemic syndromePediatr Nephrol200419441942514986082
  • ClarkWFRockGABuskardNTherapeutic plasma exchange: an update from the Canadian Apheresis GroupAnn Intern Med1999131645346210498563
  • NorisMCaprioliJBresinERelative role of genetic complement abnormalities in sporadic and familial aHUS and their impact on clinical phenotypeClin J Am Soc Nephrol20105101844185920595690
  • RuebnerRLKaplanBSCopelovitchLA time for reappraisal of “atypical” hemolytic uremic syndrome: should all patients be treated the same?Eur J Pediatr2012171101519152522673930
  • BaskinEGullerogluKKantarABayrakciUOzkayaOSuccess of eculizumab in the treatment of atypical hemolytic uremic syndromePediatr Nephrol201530578378925384530
  • Fremeaux-BacchiVPathophysiology of atypical hemolytic uremic syndrome. Ten years of progress, from laboratory to patientBiol Aujourdhui20132074231240 French24594571
  • SzarvasNSzilagyiATasicVFirst-line therapy in atypical hemolytic uremic syndrome: consideration on infants with a poor prognosisItal J Pediatr20144010125496981
  • CatalandSRHolersVMGeyerSYangSWuHMBiomarkers of terminal complement activation confirm the diagnosis of aHUS and differentiate aHUS from TTPBlood2014123243733373824695849
  • CaprioliJNorisMBrioschiSGenetics of HUS: the impact of MCP, CFH, and IF mutations on clinical presentation, response to treatment, and outcomeBlood200610841267127916621965
  • HeinenSPlutheroFGvan EimerenVFQuagginSELichtCMonitoring and modeling treatment of atypical hemolytic uremic syndromeMol Immunol2013541848823220071
  • MichonBMoghrabiAWinikoffRComplications of apheresis in childrenTransfusion200747101837184217880609
  • DeSWatersAMSegalAOTrautmannAHarveyEALichtCSevere atypical HUS caused by CFH S1191L – case presentation and review of treatment optionsPediatr Nephrol20102519710419856002
  • LegendreCMLichtCMuusPTerminal complement inhibitor eculizumab in atypical hemolytic-uremic syndromeN Engl J Med2013368232169218123738544
  • LichtCGreenbaumLAMuusPEfficacy and safety of eculizumab in atypical hemolytic uremic syndrome from 2-year extensions of phase 2 studiesKidney Int20158751061107325651368
  • Alexion Pharmaceuticals [webpage on the Internet]An open-label, multi-center clinical trial of eculizumab in adult patients with atypical hemolytic-uremic syndrome Available from: http://clinicaltrials.gov/ct2/show/NCT01194973?term=nct01194973&rank=1. NLM identifier: NCT01194973Accessed August 7, 2013
  • CofiellRKukrejaABedardKEculizumab reduces complement activation, inflammation, endothelial damage, thrombosis, and renal injury markers in aHUSBlood2015125213253326225833956
  • ArdissinoGWally OssolaMBafferoGMRigottiACugnoMEculizumab for atypical hemolytic uremic syndrome in pregnancyObstet Gynecol20131222 pt 248748923884270
  • ArdissinoGTelFTestaSSkin Involvement in atypical hemolytic uremic syndromeAm J Kidney Dis201463465265524290245
  • DavidRHochberg-KleinSAmerRResolution of ocular involvement with systemic eculizumab therapy in atypical hemolytic-uremic syndromeEye (Lond)201327899799823722719
  • FakhouriFDelmasYProvotFInsights from the use in clinical practice of eculizumab in adult patients with atypical hemolytic uremic syndrome affecting the native kidneys: an analysis of 19 casesAm J Kidney Dis2014631404824021908
  • MussoniMPVenezianoFABoettiLInnovative therapeutic approach: sequential treatment with plasma exchange and eculizumab in a pregnant woman affected by atypical hemolytic-uremic syndromeTransfus Apher Sci201451213413625294113
  • NguyenMHMathewJJDenunzioTMCarmichaelMGDiagnosis of atypical hemolytic uremic syndrome and response to eculizumab therapyHawaii J Med Public Health2014739 suppl 1222425285252
  • OhanianMCableCHalkaKEculizumab safely reverses neurologic impairment and eliminates need for dialysis in severe atypical hemolytic uremic syndromeClin Pharmacol2011351222287852
  • PoveyHVundruRJungleeNJibaniMRenal recovery with eculizumab in atypical hemolytic uremic syndrome following prolonged dialysisClin Nephrol201482532633123557793
  • RafiqATariqHAbbasNShenoyRAtypical hemolytic-uremic syndrome: a case report and literature reviewAm J Case Rep20151610911425708146
  • RigothierCDelmasYRoumeninaLTDistal angiopathy and atypical hemolytic uremic syndrome: clinical and functional properties of an anti-factor H IgAlambda antibodyAm J Kidney Dis201566233133626015278
  • SalemGFlynnJMCatalandSRProfound neurological injury in a patient with atypical hemolytic uremic syndromeAnn Hematol201392455755823139014
  • Sengul SamanciNAyerMErgenAOzturkSAn effective treatment of atypical hemolytic uremic syndrome with plasma exchange and eculizumab: a case reportTransfus Apher Sci201552331431625634788
  • SevincMBasturkTSahutogluTPlasma resistant atypical hemolytic uremic syndrome associated with a CFH mutation treated with eculizumab: a case reportJ Med Case Rep2015919225925370
  • ThajudeenBSussmanABracamonteEA case of atypical hemolytic uremic syndrome successfully treated with eculizumabCase Rep Nephrol Urol20133213914624570684
  • TsaiHMKuoEEculizumab therapy leads to rapid resolution of thrombocytopenia in atypical hemolytic uremic syndromeAdv Hematol2014201417 Article ID 295323
  • ZschiedrichSPragerEPKuehnEWSuccessful treatment of the postpartum atypical hemolytic uremic syndrome with eculizumabAnn Intern Med201315917623817717
  • Al-AkashSIAlmondPSSavellVHJrGharaybehSIHogueCEculizumab induces long-term remission in recurrent post-transplant HUS associated with C3 gene mutationPediatr Nephrol201126461361921125405
  • AricetaGArrizabalagaBAguirreMMorteruelELopez-TrascasaMEculizumab in the treatment of atypical hemolytic uremic syndrome in infantsAm J Kidney Dis201259570771022196848
  • AzukaitisKLoiratCMalinaMAdomaitieneIJankauskieneAMacrovascular involvement in a child with atypical hemolytic uremic syndromePediatr Nephrol20142971273127724352218
  • BekassyZDKristofferssonACCronqvistMEculizumab in an anephric patient with atypical haemolytic uraemic syndrome and advanced vascular lesionsNephrol Dial Transplant201328112899290724009284
  • BelingheriMPossentiITelFCryptic activity of atypical hemolytic uremic syndrome and eculizumab treatmentPediatrics20141336e1769e177124843058
  • BesbasNGulhanBKarpmanDNeonatal onset atypical hemolytic uremic syndrome successfully treated with eculizumabPediatr Nephrol201328115515822956028
  • CayciFSCakarNHancerVSUncuNAcarBGurGEculizumab therapy in a child with hemolytic uremic syndrome and CFI mutationPediatr Nephrol201227122327233122903728
  • ChristmannMHansenMBergmannCSchwabeDBrandJSchneiderWEculizumab as first-line therapy for atypical hemolytic uremic syndromePediatrics20141336e1759e176324843059
  • CoppoRPeruzziLAmoreADramatic effects of eculizumab in a child with diffuse proliferative lupus nephritis resistant to conventional therapyPediatr Nephrol201530116717225173358
  • CullinanNGormanKMRiordanMWaldronMGoodshipTHJAwanACase report: benefits and challenges of long-term eculizumab in atypical hemolytic uremic syndromePediatrics20151356e1506e150925941307
  • Diamante ChiodiniBDavinJCCorazzaFEculizumab in anti-factor h antibodies associated with atypical hemolytic uremic syndromePediatrics20141336e1764e176824843055
  • DorresteijnEMvan de KarNCCransbergKEculizumab as rescue therapy for atypical hemolytic uremic syndrome with normal platelet countPediatr Nephrol20122771193119522399074
  • GiordanoMCastellanoGMessinaGPreservation of renal function in atypical hemolytic uremic syndrome by eculizumab: a case reportPediatrics20121305e1385e138823027168
  • GruppoRARotherRPEculizumab for congenital atypical hemolytic-uremic syndromeN Engl J Med2009360554454619179329
  • GullerogluKFidanKHancerVSBayrakciUBaskinESoylemezogluONeurologic involvement in atypical hemolytic uremic syndrome and successful treatment with eculizumabPediatr Nephrol201328582783023389237
  • HisanoMAshidaANakanoEAutoimmune-type atypical hemolytic uremic syndrome treated with eculizumab as first-line therapyPediatr Int201557231331725868950
  • HuHNagraAHaqMRGilbertRDEculizumab in atypical haemolytic uraemic syndrome with severe cardiac and neurological involvementPediatr Nephrol20142961103110624317637
  • LapeyraqueALFremeaux-BacchiVRobitaillePEfficacy of eculizumab in a patient with factor-H-associated atypical hemolytic uremic syndromePediatr Nephrol201126462162421161283
  • MacheCJAcham-RoschitzBFremeaux-BacchiVComplement inhibitor eculizumab in atypical hemolytic uremic syndromeClin J Am Soc Nephrol2009481312131619556379
  • MalinaMGulatiABaggaAMajidMASimkovaESchaeferFPeripheral gangrene in children with atypical hemolytic uremic syndromePediatrics20131311e331e33523230076
  • MichauxKBacchettaJJavouheyECochatPFremaux-BacchiVSellier-LeclercALEculizumab in neonatal hemolytic uremic syndrome with homozygous factor H deficiencyPediatr Nephrol201429122415241925149852
  • NooneDAl-MatrafiJTinckamKAntibody mediated rejection associated with complement factor h-related protein 3/1 deficiency successfully treated with eculizumabAm J Transplant20121292546255322681773
  • OhtaTUrayamaKTadaYEculizumab in the treatment of atypical hemolytic uremic syndrome in an infant leads to cessation of peritoneal dialysis and improvement of severe hypertensionPediatr Nephrol201530460360825318620
  • Roman-OrtizEMendizabalOSPintoSLopez-TrascasaMSanchez-CorralPRodriguez deCSEculizumab long-term therapy for pediatric renal transplant in aHUS with CFH/CFHR1 hybrid genePediatr Nephrol201429114915323982707
  • SchalkGKirschfinkMWehlingCA complicated case of atypical hemolytic uremic syndrome with frequent relapses under eculizumabPediatr Nephrol20153061039104225752761
  • SharmaSPradhanMMeyersKELe PalmaKLaskinBLNeonatal atypical hemolytic uremic syndrome from a factor H mutation treated with eculizumabClin Nephrol201584918118525816809
  • TschumiSGuggerMBucherBSRiedlMSimonettiGDEculizumab in atypical hemolytic uremic syndrome: long-term clinical course and histological findingsPediatr Nephrol201126112085208821877169
  • VaisbichMHHenriquesLSWatanabeAEculizumab for the treatment of atypical hemolytic uremic syndrome: case report and revision of the literatureJ Bras Nefrol201335323724124100744
  • VilaltaRLaraEMadridALong-term eculizumab improves clinical outcomes in atypical hemolytic uremic syndromePediatr Nephrol201227122323232622890512
  • NorisMRemuzziGManaging and preventing atypical hemolytic uremic syndrome recurrence after kidney transplantationCurr Opin Nephrol Hypertens201322670471224076560
  • Le QuintrecMZuberJMoulinBComplement genes strongly predict recurrence and graft outcome in adult renal transplant recipients with atypical hemolytic and uremic syndromeAm J Transplant201313366367523356914
  • SalandJLiver-kidney transplantation to cure atypical HUS: still an option post-eculizumab?Pediatr Nephrol201429332933224362724
  • ZuberJLe QuintrecMKridSEculizumab for atypical hemolytic uremic syndrome recurrence in renal transplantationAm J Transplant201212123337335422958221
  • NurnbergerJPhilippTWitzkeOEculizumab for atypical hemolytic-uremic syndromeN Engl J Med2009360554254419179328
  • ChateletVLobbedezTFremeaux-BacchiVFicheuxMRyckelynckJPHurault de LignyBEculizumab: safety and efficacy after 17 months of treatment in a renal transplant patient with recurrent atypical hemolytic-uremic syndrome: case reportTransplant Proc201042104353435521168697
  • LegaultDJBoelkinsMRSuccessful treatment of aHUS recurrence and arrest of plasma exchange resistant TMA post-renal transplantation with the terminal complement inhibitor eculizumab [abstract 2421]Blood2009114suppl2421
  • AkchurinODograSKaskelFJanDGreensteinSDel RioMPreemptive use of eculizumab for living-donor kidney transplantation in a child with atypical hemolytic uremic syndromeEinstein J Biol Med2015302225
  • AlasfarSAlachkarNAtypical hemolytic uremic syndrome post-kidney transplantation: two case reports and review of the literatureFront Med (Lausanne)201415225593925
  • KridSRoumeninaLTBeuryDRenal transplantation under prophylactic eculizumab in atypical hemolytic uremic syndrome with CFH/CFHR1 hybrid proteinAm J Transplant20121271938194422494769
  • MallettAHughesPSzerJAtypical haemolytic uraemic syndrome treated with the complement inhibitor eculizumab: the experience of the Australian compassionate access cohortIntern Med J201545101054106526247170
  • MatarDNaqviFRacusenLCCarter-MonroeNMontgomeryRAAlachkarNAtypical hemolytic uremic syndrome recurrence after kidney transplantationTransplantation201498111205121224933457
  • NesterCStewartZMyersDPre-emptive eculizumab and plasmapheresis for renal transplant in atypical hemolytic uremic syndromeClin J Am Soc Nephrol2011661488149421617085
  • ParikovaAFronekJPViklickyOLiving-donor kidney transplantation for atypical haemolytic uremic syndrome with pre-emptive eculizumab useTranspl Int201528336636925256057
  • PelicanoMBde CordobaSRDiekmannFAnti-C5 as prophylactic therapy in atypical hemolytic uremic syndrome in living-related kidney transplantationTransplantation2013964e26e2923958887
  • RanchDCrowtherBArarMAssanasenCProphylactic eculizumab for kidney transplantation in a child with atypical hemolytic uremic syndrome due to complement factor H mutationPediatr Transplant2014186E185E18924931815
  • TranHChaudhuriAConcepcionWGrimmPCUse of eculizumab and plasma exchange in successful combined liver-kidney transplantation in a case of atypical HUS associated with complement factor H mutationPediatr Nephrol201429347748024221349
  • WeitzMAmonOBasslerDKoenigsrainerANadalinSProphylactic eculizumab prior to kidney transplantation for atypical hemolytic uremic syndromePediatr Nephrol20112681325132921556717
  • XieLNesterCMReedAIZhangYSmithRJThomasCPTailored eculizumab therapy in the management of complement factor H-mediated atypical hemolytic uremic syndrome in an adult kidney transplant recipient: a case reportTransplant Proc201244103037304023195022
  • ZimmerhacklLBHoferJCortinaGProphylactic eculizumab after renal transplantation in atypical hemolytic-uremic syndromeN Engl J Med2010362181746174820445192
  • AlachkarNBagnascoSMMontgomeryRAEculizumab for the treatment of two recurrences of atypical hemolytic uremic syndrome in a kidney allograftTranspl Int2012258e93e9522591029
  • CanigralCMoscardoFCastroCEculizumab for the treatment of pregnancy-related atypical hemolytic uremic syndromeAnn Hematol20149381421142224306089
  • ChandranSBaxter-LoweLOlsonJLTomlanovichSJWebberAEculizumab for the treatment of de novo thrombotic microangiopathy post simultaneous pancreas-kidney transplantation – a case reportTransplant Proc20114352097210121693335
  • De Sousa AmorimEBlascoMQuintanaLSoleMde CordobaSRCampistolJMEculizumab in pregnancy-associated atypical hemolytic uremic syndrome: insights for optimizing managementJ Nephrol201528564164525712233
  • GilbertRDFowlerDJAngusEHardySAStanleyLGoodshipTHEculizumab therapy for atypical haemolytic uraemic syndrome due to a gain-of-function mutation of complement factor BPediatr Nephrol20132881315131823624872
  • KourouklarisAIoannouKAthanasiouIPanagidouADemetriouKZavrosMPostpartum thrombotic microangiopathy revealed as atypical hemolytic uremic syndrome successfully treated with eculizumab: a case reportJ Med Case Rep2014830725219386
  • KransdorfEPKittlesonMMKobashigawaJAAtypical hemolytic-uremic syndrome immediately after heart transplantationJ Heart Lung Transplant201433666466524630405
  • LarreaCFCofanFOppenheimerFCampistolJMEscolarGLozanoMEfficacy of eculizumab in the treatment of recurrent atypical hemolytic-uremic syndrome after renal transplantationTransplantation201089790390420386298
  • PuJJSidoASuccessful discontinuation of eculizumab therapy in a patient with aHUSAnn Hematol20149381423142524346709
  • WilsonCTorpeyNJaquesBSuccessful simultaneous liver-kidney transplant in an adult with atypical hemolytic uremic syndrome associated with a mutation in complement factor HAm J Kidney Dis201158110911221601332
  • ZlamyMHoferJEliasJImmunogenicity of meningococcus C vaccination in a patient with atypical hemolytic uremic syndrome (aHUS) on eculizumab therapyPediatr Transplant2012166E246E25021967634
  • SajanTVinaySSonuNAlanPHow atypical can atypical hemolytic uremic syndrome be?Clin Case Rep201422575925356245
  • VellankiVSBargmanJMAspergillus niger peritonitis in a peritoneal dialysis patient treated with eculizumabRen Fail201436463163324512095
  • HayesWTschumiSLingSCFeberJKirschfinkMLichtCEculizumab hepatotoxicity in pediatric aHUSPediatr Nephrol201530577578125416628
  • HallstensenRFBergsethGFossSEculizumab treatment during pregnancy does not affect the complement system activity of the newbornImmunobiology2015220445245925468724
  • CugnoMGualtierottiRPossentiIComplement functional tests for monitoring eculizumab treatment in patients with atypical hemolytic uremic syndromeJ Thromb Haemost20141291440144824853860
  • ArdissinoGTestaSPossentiIDiscontinuation of eculizumab maintenance treatment for atypical hemolytic uremic syndrome: a report of 10 casesAm J Kidney Dis201464463363724656451
  • CarrRCatalandSRRelapse of aHUS after discontinuation of therapy with eculizumab in a patient with aHUS and factor H mutationAnn Hematol201392684584623154867
  • ArdissinoGPossentiITelFTestaSSalardiSLadisaVDiscontinuation of eculizumab treatment in atypical hemolytic uremic syndrome: an updateAm J Kidney Dis201566117217326111906
  • WetzelsJFvan de KarNCDiscontinuation of eculizumab maintenance treatment for atypical hemolytic uremic syndromeAm J Kidney Dis201565234225616634
  • Cornec-Le GallEDelmasYDe ParscauLAdult-onset eculizumab-resistant hemolytic uremic syndrome associated with cobalamin C deficiencyAm J Kidney Dis201463111912324210589
  • CheungpasitpornWLeungNSethiSGertzMAFervenzaFCRefractory atypical hemolytic uremic syndrome with monoclonal gammopathy responsive to bortezomib-based therapyClin Nephrol201583636336925345382
  • NishimuraJYamamotoMHayashiSGenetic variants in C5 and poor response to eculizumabN Engl J Med2014370763263924521109