ArticlePediatric nephrology (Berlin, Germany)2012
Eculizumab therapy in a child with hemolytic uremic syndrome and CFI mutation.
Article in Pediatric nephrology (Berlin, Germany), 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
24 citing papers in PubMed, 39 citations in OpenAlex.
- Trial
- Efficacy and safety of eculizumab in atypical hemolytic uremic syndrome from 2-year extensions of phase 2 studies.Kidney international · 2015Trial
- Article
- Complement gene variant effect on relapse of complement-mediated thrombotic microangiopathy after eculizumab cessation.Blood advances · 2023Article
- Fatal thrombotic microangiopathy case following adeno-associated viral SMN gene therapy.Blood advances · 2022Article
- Atypical haemolytic uraemic syndrome: a case of rare genetic mutation.BMJ case reports · 2021Article
- Rare Genetic Variants in Jewish Patients Suffering from Age-Related Macular Degeneration.Genes · 2019Article
- Eculizumab prevents thrombotic microangiopathy in patients with atypical haemolytic uraemic syndrome in a long-term observational study.Clinical kidney journal · 2019Article
- Clinical Relapses of Atypical HUS on Eculizumab: Clinical Gap for Monitoring and Individualised Therapy.Case reports in nephrology · 2018Article
- Current evidence on the discontinuation of eculizumab in patients with atypical haemolytic uraemic syndrome.Clinical kidney journal · 2017Article
- Rare genetic variant in the CFB gene presenting as atypical hemolytic uremic syndrome and immune complex diffuse membranoproliferative glomerulonephritis, with crescents, successfully treated with eculizumab.Pediatric nephrology (Berlin, Germany) · 2017Article
- The complement system in age-related macular degeneration: A review of rare genetic variants and implications for personalized treatment.Molecular immunology · 2017Review
- First-Line, Early and Long-Term Eculizumab Therapy in Atypical Hemolytic Uremic Syndrome: A Case Series in Pediatric Patients.Paediatric drugs · 2016Article
- Review
- The genetic fingerprint of susceptibility for transplant-associated thrombotic microangiopathy.Blood · 2016Article
- An international consensus approach to the management of atypical hemolytic uremic syndrome in children.Pediatric nephrology (Berlin, Germany) · 2016Review
- Critical appraisal of eculizumab for atypical hemolytic uremic syndrome.Journal of blood medicine · 2016Review
- Rare genetic variants in the CFI gene are associated with advanced age-related macular degeneration and commonly result in reduced serum factor I levels.Human molecular genetics · 2015Article
- Podocyte dysfunction in atypical haemolytic uraemic syndrome.Nature reviews. Nephrology · 2015Review
- Postpartum thrombotic microangiopathy revealed as atypical hemolytic uremic syndrome successfully treated with eculizumab: a case report.Journal of medical case reports · 2014Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundHemolytic uremic syndrome (HUS) is the most common cause of acute renal failure in childhood. It usually occurs after a prodromal episode of diarrhea and it leads to significant morbidity and mortality during the acute phase. However, cases that start as diarrhea-positive HUS whose renal function fail to recover should be screened for genetic disorders of the complement system, which is called atypical HUS (aHUS). CASE-DIAGNOSIS/TREATMENT: We herein report a 10-year-old girl, who initially came with bloody diarrhea and had features of HUS with delayed renal and hematological recovery despite plasma therapy. Eculizumab (600 mg/week) was initiated on day 15 for atypical presentation and later a complement factor I (CFI) mutation was detected. The girl recovered diuresis within 24 h and after the third eculizumab infusion, hemoglobin, platelet, and C3 levels normalized; renal function improved; and proteinuria completely disappeared in 2 weeks.
conclusionIt is our belief that eculizumab can be the treatment of choice in children who have plasma exchange-refractory HUS with defective regulation of the alternative complement pathway.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.