Eculizumab for the treatment of atypical hemolytic uremic syndrome - Case report and revision of the literature

Vaisbich, María Helena; Henriques, Luciana dos Santos; Watanabe, Andréia; Pereira, Lilian Elgalise Techio; Metran, Camila Cardoso; Malheiros, Denise Avancini; Modanez, Flávia; Silva, João Domingos Montoni da; Vieira, Simone; Macedo, Ana Catarina Lunz; Massarope, Bianca; Furusawa, Erika Arai; Schvartsman, Benita Galassi Soares

doi:10.5935/0101-2800.20130037

Cited by 7 publications

(3 citation statements)

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“…In the second case, eculizumab therapy was introduced 15 days after the initial suspicion of aHUS. Eculizumab, a human monoclonal antibody that stops the activation of the C5 complement component and the final complement formation, has recently been shown to be effective in the treatment of aHUS [ 28 – 30 ]. It helps in the recovery of kidney function and can completely change the prognosis of this potentially catastrophic disorder [ 31 ].…”

Section: Case Discussionmentioning

confidence: 99%

Thrombotic microangiopathy associated with arboviral infection: Report of 3 cases

et al. 2021

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Dengue fever and chikungunya are viral diseases that have spread rapidly throughout the world in recent decades. The occurrence of complications is well known, including prerenal acute kidney injury (AKI), which is usually thought to be caused by dehydration and fluid loss. Thrombotic microangiopathy (TMA) is an uncommon aggravation of dengue fever and chikungunya, with only a few cases described in the medical literature. The aim of this study is to present 3 cases of TMA associated with arboviral infection. Three patients with clinical history, laboratory test, and kidney biopsy results compatible with TMA were selected for the study, 2 of whom had a serological diagnosis of dengue fever and 1 of chikungunya. The 3 patients were followed up at the Federal University of Maranhão Hospital’s Nephrology Service in 2018. A targeted gene panel sequencing (TGPS) plus multiple to atypical hemolytic uremic syndrome (aHUS) multiplex ligation–dependent probe amplification (MLPA) was performed in 2 of the patients and revealed in the patient 1 a heterozygous pathogenic variant in the gene THBD, as well as heterozygous deletions in CFH, CFHR1, and CFHR3. In the patient 2, there were heterozygous pathogenic variant in the genes CFI and CFB, in addition to heterozygous deletions in the genes CFHR1 and CFHR3. Both received treatment with eculizumab and undergone recovery of renal function. The third patient had TMA not classified as either aHUS or thrombotic thrombocytopenic purpura (TTP); he abandoned the treatment and returned to the service after 2 years for a dialysis emergency. Patients with arboviral infectious disease and changes that suggest TMA should have appropriate support to establish early diagnosis and useful treatment.

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Section: Case Discussionmentioning

confidence: 99%

Thrombotic microangiopathy associated with arboviral infection: Report of 3 cases

et al. 2021

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“…This suggests that early initiation of eculizumab therapy may be more effective in conservation of renal function and recovery from reversible insult [31]. More cases of success continue to be reported with eculizumab in patients with aHUS before end-stage renal disease and even after kidney transplantation [28,32–35]. …”

Section: Discussionmentioning

confidence: 99%

Atypical Hemolytic-Uremic Syndrome: A Case Report and Literature Review

et al. 2015

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Patient: Female, 59Final Diagnosis: Atyipcal hemolytic uremic syndromeSymptoms: Delirium • headacheMedication: —Clinical Procedure: —Specialty: HematologyObjective:Rare diseaseBackground:Atypical hemolytic uremic syndrome (aHUS) is a rare disease characterized by hemolysis, thrombocytopenia, and renal dysfunction. It is a disease related to genetic mutations in the alternative complement pathway and has a distinct pathophysiology but is difficult to differentiate from other thrombotic microangiopathies.Case Report:We present a case of a 59-year-old female patient who presented with accelerated hypertension, acute renal failure, hemolysis, and encephalopathy. She was managed with antihypertensive medication, but her encephalopathy did not improve. Evaluation resulted in our impression of the disease being atypical hemolytic-uremic syndrome. The patient continued to be managed with good blood pressure control and later was started on eculizumab, but evaluation of response to therapy was hindered by the patient’s non-compliance with therapy and follow-up appointments.Conclusions:We have a very limited understanding of the genetics and epidemiology of atypical HUS, and the overlapping clinical features sometimes delay diagnosis and initiation of appropriate treatment of this rare disease.

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“…Eculizumab was first used with success in patients with PNH. Nowadays several dozen retrospective observations of children and adults treated with eculizumab for aHUS in native or transplanted kidneys have been published [Gruppo and Rother, 2009; Nürnberger et al 2009; Chatelet et al 2009; Davin et al 2010; Zimmerhackl et al 2010; Heyne et al 2011; Nester et al 2011; Weitz et al 2011; Wilson et al 2011; Ariceta et al 2012; Dorresteijn et al 2012; Ardissino et al 2013; Cañigral et al 2013; Gilbert et al 2013; Gulleroglu et al 2013; Legendre et al 2013b; Pelicano et al 2013; Povey et al 2013; Reuter et al 2013; Sinibaldi et al 2013; Thajudeen et al 2013; Vaisbich et al 2013; Belingheri et al 2014; Chiodini et al 2014; Christmann et al 2014; Fakhouri et al 2014; Green et al 2014; Román-Ortiz et al 2014; Tran et al 2014]. All described success on renal, neurological, cutaneous and myocardial lesions, in all kind of mutations and also when mutations were not identified, and in anti-CFH antibodies mediated HUS.…”

Section: Treatment Of Ahusmentioning

confidence: 99%

Advances and challenges in the management of complement-mediated thrombotic microangiopathies

Davin

Kar²

2015

Therapeutic Advances in Hematology

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Complement activation plays a major role in several renal pathophysiological conditions. The three pathways of complement lead to C3 activation, followed by the formation of the anaphylatoxin C5a and the terminal membrane attack complex (MAC) in blood and at complement activating surfaces, lead to a cascade of events responsible for inflammation and for the induction of cell lysis. In case of ongoing uncontrolled complement activation, endothelial cells activation takes place, leading to events in which at the end thrombotic microangiopathy can occur. Atypical haemolytic uremic syndrome (aHUS) is a thrombotic microangiopathy characterized by excessive complement activation on the surface of the microcirculation. It is a severe, rare disease which leads to end-stage renal failure (ESRF) and/or to death in more than 50% of patients without treatment. In the first decade of the second millennium, huge progress in understanding the aetiology of this disease was made, which paved the way to better treatment. First, protocols of plasma therapy for treatment, prevention of relapses and for renal transplantation in those patients were set up. Secondly, in some severe cases, combined kidney and liver transplantation was reported. Finally, at the end of this decade, the era of complement inhibitors, as anti-C5 monoclonal antibody (anti-C5 mAb) began. The past five years have seen growing evidence of the favourable effect of anti-C5 mAb in aHUS which has made this drug the first-line treatment in this disease. The possible complication of meningococcal infection needs appropriate vaccination before its use. Unfortunately, the worldwide use of anti-C5 mAb is limited by its very high price. In the future, extension of indications for anti-C5 mAb use, the elaboration of generics and of mAbs directed towards other complement factors of the terminal pathway of the complement system might succeed in reducing the cost of this new valuable therapeutic approach and render it available worldwide for patients from all social classes.

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Eculizumab for the treatment of atypical hemolytic uremic syndrome - Case report and revision of the literature

Cited by 7 publications

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Thrombotic microangiopathy associated with arboviral infection: Report of 3 cases

Thrombotic microangiopathy associated with arboviral infection: Report of 3 cases

Atypical Hemolytic-Uremic Syndrome: A Case Report and Literature Review

Advances and challenges in the management of complement-mediated thrombotic microangiopathies

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