ReviewTherapeutics and clinical risk management2016
Eculizumab in the management of paroxysmal nocturnal hemoglobinuria: patient selection and special considerations.
Review in Therapeutics and clinical risk management, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers, 1 of them a synthesis 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
26 citing papers in PubMed, 1 synthesis or guideline pooled it, 33 citations in OpenAlex.
- Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Expert Opinion from Central Europe on Special Patient Populations.Advances in therapy · 2023Pooled it
- Iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria: a 2-cohort open-label proof-of-concept study.Blood advances · 2022Trial
- Article
- The Path to Accessible Care: Development and Impact of Eculizumab Biosimilars for Paroxysmal Nocturnal Hemoglobinuria and Atypical Hemolytic Uremic Syndrome.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2025Review
- Advances in Complement Inhibition Therapies for Paroxysmal Nocturnal Hemoglobinuria and Autoimmune Hemolytic Disorders.Journal of blood medicine · 2025Review
- Enhanced complement activation and MAC formation accelerates severe COVID-19.Cellular and molecular life sciences : CMLS · 2024Article
- Pegcetacoplan for Treating Paroxysmal Nocturnal Haemoglobinuria: An Evidence Review Group Perspective of a NICE Single Technology Appraisal.PharmacoEconomics - open · 2023Review
- Article
- Targeting the Complement-Sphingolipid System in COVID-19 and Gaucher Diseases: Evidence for a New Treatment Strategy.International journal of molecular sciences · 2022Review
- Real-World Eculizumab Dosing Patterns Among Patients with Paroxysmal Nocturnal Hemoglobinuria in a US Population.ClinicoEconomics and outcomes research : CEOR · 2022Article
- Rescue of Glycosylphosphatidylinositol-Anchored Protein Biosynthesis Using Synthetic Glycosylphosphatidylinositol Oligosaccharides.ACS chemical biology · 2021Article
- Infectious Complications in Autoimmune Hemolytic Anemia.Journal of clinical medicine · 2021Review
- Small hepatic veins Budd-Chiari syndrome and paroxysmal nocturnal hemoglobinuria - The association of two rare entities: a case report.Pathologica · 2020Article
- Multi-component meningococcal serogroup B (MenB)-4C vaccine induces effective opsonophagocytic killing in children with a complement deficiency.Clinical and experimental immunology · 2019Article
- The Extended Use of Eculizumab in Pregnancy and Complement Activation⁻Associated Diseases Affecting Maternal, Fetal and Neonatal Kidneys-The Future Is Now?Journal of clinical medicine · 2019Review
- Differential miRNA expression profile and proteome in plasma exosomes from patients with paroxysmal nocturnal hemoglobinuria.Scientific reports · 2019Article
- Therapeutic challenges in pregnant women with paroxysmal nocturnal hemoglobinuria: A case report.Medicine · 2018Article
- Review
- Nephrotic-range proteinuria and brown urine in an 8-year-old girl: Answers.Pediatric nephrology (Berlin, Germany) · 2018Article
- The MFHR1 Fusion Protein Is a Novel Synthetic Multitarget Complement Inhibitor with Therapeutic Potential.Journal of the American Society of Nephrology : JASN · 2018Article
Corrections and comments
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Authors and funding
3 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Paroxysmal nocturnal hemoglobinuria (PNH) is a nonmalignant clonal disorder resulting from somatic mutation in the PIG-A gene leading to a deficiency of the membrane-anchoring molecule glycosylphosphatidylinositol. The lack of expression of two glycosylphosphatidylinositol-anchored proteins involved in the regulation of the complement system renders PNH erythrocytes susceptible to complement-mediated lysis. Clinical manifestations include thromboembolic disease, chronic kidney injury, pulmonary hypertension, smooth muscle dysfunction, and chronic hemolysis. Until recently, treatment was mainly supportive with most patients suffering from significant morbidity and shortened survival compared to age-matched controls. The development of eculizumab, a humanized monoclonal antibody directed against the terminal complement protein C5, has resulted in dramatic improvements of survival and reduction in complications. In this paper, we review some special considerations pertaining to the use of eculizumab for PNH.
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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.