Eculizumab in neonatal hemolytic uremic syndrome with homozygous factor H deficiency

Michaux, Katell; Bacchetta, Justine; Javouhey, Étienne; Cochat, Pierre; Frémaux-Bacchi, V.; Sellier‐Leclerc, Anne‐Laure

doi:10.1007/s00467-014-2933-1

Cited by 18 publications

(17 citation statements)

References 15 publications

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“…Few case reports and series have also demonstrated long-term efficacy and safety of Eculizumab as first line treatment in neonates and infants who suffered from aHUS. Similar to our patient, neonates and infants in the studies also showed complete recovery of the renal function after initiation of Ezulizumab [16, 17]. …”

Section: Discussionsupporting

confidence: 86%

Atypical hemolytic uremic syndrome in first trimester pregnancy successfully treated with eculizumab

et al. 2017

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Background Atypical hemolytic uremic syndrome is a rare disorder which is known to cause acute thrombotic microangiopathy during pregnancy with poor maternal and fetal outcomes. Atypical hemolytic uremic syndrome is caused mostly by dysregulation of alternative complement pathway secondary to genetic mutations. Most of the cases reported have been in the post-partum period. We report a rare case of a patient who presents with thrombotic microangiopathy in the first trimester of her eleventh pregnancy and was successfully treated with eculizumab.Case presentationA 30-year-old woman presented at 10 weeks of gestation with hypertension, hemolytic anemia, thrombocytopenia, and acute kidney injury, consistent with thrombotic microangiopathy. She was managed initially with daily plasmapheresis. However, her kidney function did not recover, requiring hemodialysis. ADAMTS13 activity was later found to be within normal limit, hence diagnosis of atypical hemolytic uremic syndrome was strongly considered at that time and she was immediately treated with anti-C5 humanized monoclonal antibody (eculizumab). The patient responded well (resolution of thrombotic microangiopathy and recovery of renal function) to eculizumab, with continued remission after discharge and successfully delivered a healthy baby at term without any peripartum complications.ConclusionEarly recognition of atypical hemolytic uremic syndrome is often difficult as several other conditions also manifest as thrombotic microangiopathy during pregnancy, causing delay in initiating appropriate treatment. Our case suggests that treatment of atypical hemolytic uremic syndrome in early trimester of pregnancy with eculizumab results in good outcome to mother and fetus.

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Section: Discussionsupporting

confidence: 86%

Atypical hemolytic uremic syndrome in first trimester pregnancy successfully treated with eculizumab

et al. 2017

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“…This new drug was reported to be safe and effective in children in several case reports and clinical trials [20-23]. In a prospective study reported by Greenbaum et al [24], 64% of 22 pediatric patients achieved complete remission after 26 weeks of treatment.…”

Section: Discussionmentioning

confidence: 99%

Atypical Hemolytic Uremic Syndrome in Children Aged <2 Years

Çakar

Özçakar

Özaltın

et al. 2018

Nephron

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Background: There are limited data on infants with atypical hemolytic uremic syndrome (aHUS). The aim of this study was to determine the clinical and laboratory features, and to evaluate treatment modalities and outcomes in infants with aHUS. Materials and Methods: Relevant data on patients with onset of aHUS at age <2 years were obtained from the Turkish Pediatric aHUS Registry. Results: Among the 146 patients included in the Registry, 53 (36%) (23 male and 30 female) were enrolled for the study. Age at disease onset was ≤1 year in 29 of the patients. In all, 21 (40%) of the patients developed neurological symptoms. Disease-causing mutations were noted in 14 (36%) of the 39 patients in which genetic analysis was performed. Plasma therapy was performed in 42 (79%) patients; eculizumab therapy was administered to treat the first episode of aHUS in 33 (62%) patients and in 5 patients as the first- line therapy. In total, 38 (72%) patients received renal replacement therapy (RRT), 3 (6%) died due to acute illness, and 4 (8%) were discharged from hospital with RRT. Follow-up visit data were available for 46 patients and the median duration was 23 months (range 3–129 months). End-stage renal disease developed only in 1 patient. Proteinuria and hypertension persisted in 17 (37%) and 20 patients (44%) respectively. Eculizumab treatment was continued in 25 of the 39 patients during the follow-up period. Conclusion: One-third of the aHUS patients had disease onset during infancy. The prognosis of this life-threatening disease seems to get better with improved treatment modalities.

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“…That eculizumab may be efficacious to rescue central nervous system involvement is suggested by nine case reports, including four in children [87,90,110,[123][124][125][126][127]. Eculizumab also appeared lifesaving in four children with myocardial involvement [83,90,92,110]. The reports of four children who developed cerebral ischemic events due to stenosis of cerebral arteries after several years on dialysis have suggested that local complement activation added to the vascular consequences of long-term dialysis may lead to such macrovascular complications [101,[128][129][130].…”

Section: Education Information Cardmentioning

confidence: 92%

An international consensus approach to the management of atypical hemolytic uremic syndrome in children

et al. 2015

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Atypical hemolytic uremic syndrome (aHUS) emerged during the last decade as a disease largely of complement dysregulation. This advance facilitated the development of novel, rational treatment options targeting terminal complement activation, e.g., using an anti-C5 antibody (eculizumab). We review treatment and patient management issues related to this therapeutic approach. We present consensus clinical practice recommendations generated by HUS International, an international expert group of clinicians and basic scientists with a focused interest in HUS. We aim to address the following questions of high relevance to daily clinical practice: Which complement investigations should be done and when? What is the importance of anti-factor H antibody detection? Who should be treated with eculizumab? Is plasma exchange therapy still needed? When should eculizumab therapy be initiated? How and when should complement blockade be monitored? Can the approved treatment schedule be modified? What approach should be taken to kidney and/or combined liver-kidney transplantation? How should we limit the risk of meningococcal infection under complement blockade therapy? A pressing question today regards the treatment duration. We discuss the need for prospective studies to establish evidence-based criteria for the continuation or cessation of anticomplement therapy in patients with and without identified complement mutations.

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Eculizumab in neonatal hemolytic uremic syndrome with homozygous factor H deficiency

Abstract: We report on the long-term efficacy and safety of eculizumab as first-line therapy in neonatal aHUS. However its use still requires optimization in terms of indications and administration (frequency, dosage).

Cited by 18 publications

References 15 publications

Atypical hemolytic uremic syndrome in first trimester pregnancy successfully treated with eculizumab

Atypical hemolytic uremic syndrome in first trimester pregnancy successfully treated with eculizumab

Atypical Hemolytic Uremic Syndrome in Children Aged <2 Years

An international consensus approach to the management of atypical hemolytic uremic syndrome in children

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