ArticleBritish journal of haematology2019
Eculizumab in paroxysmal nocturnal haemoglobinuria and atypical haemolytic uraemic syndrome: 10-year pharmacovigilance analysis.
Article in British journal of haematology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 125 papers, 5 of them syntheses that pooled it.
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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.
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Who cites it
125 citing papers in PubMed, 5 syntheses or guidelines pooled it, 231 citations in OpenAlex.
- Consensus Recommendations for the Diagnosis and Treatment of Neuromyelitis Optica Spectrum Disorders (NMOSD): The MENACTRIMS Guidelines.CNS drugs · 2026Guideline
- Incidence of infection associated with eculizumab: a meta-analysis of 9 randomized controlled trials.Frontiers in pharmacology · 2025Pooled it
- Therapeutic Targeting of the Complement System: From Rare Diseases to Pandemics.Pharmacological reviews · 2021Pooled it
- Interventions for atypical haemolytic uraemic syndrome.The Cochrane database of systematic reviews · 2021Pooled it
- A systematic review of the role of eculizumab in systemic lupus erythematosus-associated thrombotic microangiopathy.BMC nephrology · 2020Pooled it
- Long-term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2-year results from two pivotal phase 3 studies.European journal of haematology · 2022Trial
- Eculizumab monotherapy for NMOSD: Data from PREVENT and its open-label extension.Multiple sclerosis (Houndmills, Basingstoke, England) · 2022Trial
- Long-Term Safety and Efficacy of Eculizumab in Aquaporin-4 IgG-Positive NMOSD.Annals of neurology · 2021Trial
- 'Minimal symptom expression' in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab.Journal of neurology · 2020Trial
- Trial
- Safety of Ravulizumab Use in Pregnancy: Insights from a Global Pharmacovigilance Analysis.Advances in therapy · 2026Article
- Review
- Complement Inhibition in the Clinic: Are We Doing Enough to Protect Patients From Infection?European journal of immunology · 2026Review
- Sutimlimab in Patients With Cold Agglutinin Disease (CAD): Results From a Managed Access Program.European journal of haematology · 2026Article
- Italian recommendations for the diagnosis and treatment of myasthenia gravis.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2026Article
- A new era of complement inhibition: are we ready for the infectious risks?Pediatric nephrology (Berlin, Germany) · 2026Article
- Efficacy analysis of iptacopan in a patient with thrombotic microangiopathy after allogeneic hematopoietic stem cell transplantation: a case report.Frontiers in immunology · 2026Article
- Beyond Terminal Blockade: A Mechanism-Based Approach to Complement Inhibitor Selection in Paroxysmal Nocturnal Hemoglobinuria.Drug design, development and therapy · 2026Review
- Disease-modifying treatments in paroxysmal nocturnal hemoglobinuria: a systematic review of economic evaluations.Cost effectiveness and resource allocation : C/E · 2025Review
- Clinical characteristics and management of paroxysmal nocturnal hemoglobinuria in the Middle East: a narrative review.Clinical and experimental medicine · 2025Review
65 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
10 authors at 7 institutions in 6 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Eculizumab is the first and only medication approved for paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) treatment. However, eculizumab safety based on long-term pharmacovigilance is unknown. This analysis summarises safety data collected from spontaneous and solicited sources from 16 March 2007 through 1 October 2016. Cumulative exposure to eculizumab was 28 518 patient-years (PY) (PNH, 21 016 PY; aHUS, 7502 PY). Seventy-six cases of meningococcal infection were reported (0·25/100 PY), including eight fatal PNH cases (0·03/100 PY). Susceptibility to meningococcal infections remained the key risk in patients receiving eculizumab. The meningococcal infection rate decreased over time; related mortality remained steady. The most commonly reported serious nonmeningococcal infections were pneumonia (11·8%); bacteraemia, sepsis and septic shock (11·1%); urinary tract infection (4·1%); staphylococcal infection (2·6%); and viral infection (2·5%). There were 434 reported cases of eculizumab exposure in pregnant women; of 260 cases with known outcomes, 70% resulted in live births. Reporting rates for solid tumours (≈0·6/100 PY) and haematological malignancies (≈0·74/100 PY) remained stable over time. No new safety signals affecting the eculizumab benefit-risk profile were identified. Continued awareness and implementation of risk mitigation protocols are essential to minimise risk of meningococcal and other Neisseria infections in patients receiving eculizumab.
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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.