ArticlePediatric nephrology (Berlin, Germany)2021
The long-acting C5 inhibitor, ravulizumab, is efficacious and safe in pediatric patients with atypical hemolytic uremic syndrome previously treated with eculizumab.
Article in Pediatric nephrology (Berlin, Germany), 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to trial NCT03131219 (A Phase 3, Open-Label, Multicenter Study of ALXN1210 in Children and Adolescents With Atypical Hemolytic Uremic Syndrome), which is not on this map. Cited by 40 papers, 2 of them syntheses that pooled it.
What it found
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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.
A Phase 3, Open-Label, Multicenter Study of ALXN1210 in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)
Who cites it
40 citing papers in PubMed, 2 syntheses or guidelines pooled it, 72 citations in OpenAlex.
- Consensus Recommendations for the Diagnosis and Treatment of Neuromyelitis Optica Spectrum Disorders (NMOSD): The MENACTRIMS Guidelines.CNS drugs · 2026Guideline
- Systematic review of atypical hemolytic uremic syndrome biomarkers.Pediatric nephrology (Berlin, Germany) · 2022Pooled it
- Evaluation of ravulizumab trough levels in pediatric complement-mediated hemolytic uremic syndrome in remission.Pediatric nephrology (Berlin, Germany) · 2026Observational
- Updates in atypical hemolytic syndrome.Current opinion in pediatrics · 2026Review
- Real-World Effectiveness of Ravulizumab Among C5 Inhibitor-Naive Patients With Atypical Hemolytic Uremic Syndrome: A Physician Panel-Based Chart Review (aHUS IMPACT Study).Kidney medicine · 2026Article
- Diagnosis and treatment of complement-mediated thrombotic microangiopathies: consensus of the Genetic Diseases Committee of the Chilean Society of Nephrology.BMC nephrology · 2026Review
- New Anticomplement Drugs in Nephrology: Mechanism and Indication.Kidney & blood pressure research · 2026Review
- Therapeutic advances in eculizumab for atypical hemolytic uremic syndrome: a narrative review.Frontiers in pharmacology · 2026Review
- Effectiveness and safety of ravulizumab for Japanese patients with atypical hemolytic uremic syndrome switched from eculizumab: an analysis of a post-marketing surveillance.Clinical and experimental nephrology · 2025Article
- Emerging role of complement system in the induction of neuroinflammation in adenylosuccinate lyase deficiency disorder.Brain, behavior, & immunity - health · 2025Review
- Effectiveness and Safety of Switching to Ravulizumab From Eculizumab in Kidney Transplant Recipients With Atypical Hemolytic Uremic Syndrome: A Global aHUS Registry Analysis.Clinical transplantation · 2025Article
- Complement System Inhibitors in Nephrology: An Update-Narrative Review.International journal of molecular sciences · 2025Review
- Atypical Hemolytic Uremic Syndrome: A Review of Complement Dysregulation, Genetic Susceptibility and Multiorgan Involvement.Journal of clinical medicine · 2025Review
- Recent Developments in Pediatric Nephrology.Journal of clinical medicine · 2025Article
- Global aHUS Registry Analysis of Patients Switching to Ravulizumab From Eculizumab.Kidney international reports · 2024Article
- Ravulizumab in Atypical Hemolytic Uremic Syndrome: An Analysis of 2-Year Efficacy and Safety Outcomes in 2 Phase 3 Trials.Kidney medicine · 2024Article
- Anti-C5 monoclonal antibody treatment showing pathological resolution of complement-mediated atypical hemolytic uremic syndrome: a case report.BMC nephrology · 2024Article
- Article
- Clinical efficacy and safety of switching from eculizumab to ravulizumab in adult patients with aHUS- real-world data.BMC nephrology · 2024Article
- Complement inhibitors in pediatric kidney diseases: new therapeutic opportunities.Pediatric nephrology (Berlin, Germany) · 2024Review
Corrections and comments
- Erratum issued
Authors and funding
8 authors at 4 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundAtypical hemolytic uremic syndrome (aHUS) is a rare, complement-mediated disease associated with poor outcomes if untreated. Ravulizumab, a long-acting C5 inhibitor developed through minimal, targeted modifications to eculizumab was recently approved for the treatment of aHUS. Here, we report outcomes from a pediatric patient cohort from the ravulizumab clinical trial (NCT03131219) who were switched from chronic eculizumab to ravulizumab treatment.
methodsTen patients received a loading dose of ravulizumab on Day 1, followed by maintenance doses administered initially on Day 15, and then, every 4-8 weeks thereafter, depending on body weight. All patients completed the initial evaluation period of 26 weeks and entered the extension period.
resultsNo patients required dialysis at any point throughout the study. The median estimated glomerular filtration rate values remained stable during the trial: 99.8 mL/min/1.73m
conclusionsOverall, treatment with ravulizumab in pediatric patients with aHUS who were previously treated with eculizumab resulted in stable kidney and hematologic parameters, with no unexpected safety concerns when administered every 4-8 weeks.
trial registrationTrial identifiers: Trial ID: ALXN1210-aHUS-312 Clinical trials.gov : NCT03131219 EudraCT number: 2016-002499-29 Graphical abstract.
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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.