ArticlePediatric nephrology (Berlin, Germany)2014
Eculizumab in neonatal hemolytic uremic syndrome with homozygous factor H deficiency.
Article in Pediatric nephrology (Berlin, Germany), 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 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
9 citing papers in PubMed, 19 citations in OpenAlex.
- Complement gene variant effect on relapse of complement-mediated thrombotic microangiopathy after eculizumab cessation.Blood advances · 2023Article
- Neonatal Atypical Hemolytic Uremic Syndrome in the Eculizumab Era.AJP reports · 2021Article
- Prophylactic amoxicillin for the prevention of meningococcal infection in infants with atypical hemolytic uremic syndrome under treatment with eculizumab: a report of two cases.CEN case reports · 2020Article
- Case Report: Neonatal Unexplained HUS Treated With Complement Inhibitor Eculizumab.Frontiers in pediatrics · 2020Article
- Atypical hemolytic uremic syndrome in first trimester pregnancy successfully treated with eculizumab.Experimental hematology & oncology · 2017Article
- 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
- Neonatal atypical hemolytic uremic syndrome from a factor H mutation treated with eculizumab.Clinical nephrology · 2015Article
- Primary disease recurrence—effects on paediatric renal transplantation outcomes.Nature reviews. Nephrology · 2015Review
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 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundNeonatal atypical hemolytic uremic syndrome (aHUS) is a rare but severe disease that is mainly due to methylmalonic aciduria or genetic complement abnormalities. Traditional management of aHUS includes plasma infusion/exchange, but in small or unstable infants, plasma exchange can be challenging because of high extracorporeal volume and difficulty to obtain an adequate venous access. The C5 complement blocker eculizumab has become a cornerstone of first-line management of aHUS due to complement deregulation in older patients. However, little data are available on its use in neonatal aHUS. CASE-DIAGNOSIS/TREATMENT: We report on an 11-day-old neonate with severe aHUS (myocardial impairment, respiratory failure, acute kidney disease requiring hemodiafiltration) due to homozygous factor-H deficiency. She received early treatment with eculizumab as first-line therapy and completely recovered within 5 days. A second dose of eculizumab was administered 7 days after the first infusion, followed by a dose every 2 weeks for 2 months and then every 3 weeks, at the same dosage (300 mg). With more than 24 months of follow-up, renal function remains normal.
conclusionsWe 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).
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.