ArticleTherapeutics and clinical risk management2009
The pathophysiology of paroxysmal nocturnal hemoglobinuria and treatment with eculizumab.
Article in Therapeutics and clinical risk management, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
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Who cites it
22 citing papers in PubMed, 41 citations in OpenAlex.
- Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria.British journal of haematology · 2013Trial
- Integrating paroxysmal nocturnal hemoglobinuria (PNH) into mainstream hematological curriculum training.HemaSphere · 2026Article
- Diffuse Large B-Cell Lymphoma Combined With Paroxysmal Nocturnal Hemoglobinuria: A Rare Case Report.Clinical case reports · 2025Article
- Paroxysmal Nocturnal Hemoglobinuria: Unraveling Its Molecular Pathogenesis and Advancing Targeted Therapeutic Strategies.Diseases (Basel, Switzerland) · 2025Review
- Model-Informed Precision Dosing of Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria.Clinical pharmacokinetics · 2025Observational
- Real-World effectiveness of eculizumab in atypical hemolytic uremic syndrome: a retrospective study from Western China.Frontiers in endocrinology · 2025Article
- The Onerous task of managing paroxysmal nocturnal hemoglobinuria in a Low resource setting: a case report. A hematologist's experience.African health sciences · 2024Article
- Article
- Cost-Utility Analysis of Ravulizumab Compared with Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria.PharmacoEconomics · 2020Article
- Management of thrombosis in paroxysmal nocturnal hemoglobinuria: a clinician's guide.Therapeutic advances in hematology · 2017Review
- Biological Drugs in Guillain-Barré Syndrome: An Update.Current neuropharmacology · 2017Review
- A Case of Fulminant Meningococcemia: It Is All in the Complement.Case reports in infectious diseases · 2017Article
- Maintenance eculizumab dose adjustment in the treatment of atypical hemolytic uremic syndrome: a case report and review of the literature.Clinical case reports · 2016Article
- Pesg PNH diagnosis, follow-up and treatment guidelines.American journal of blood research · 2016Review
- Long Standing Eculizumab Treatment without Anticoagulant Therapy in High-Risk Thrombogenic Paroxysmal Nocturnal Hemoglobinuria.Hematology reports · 2015Article
- Eculizumab treatment for paroxysmal nocturnal haemoglobinuria in a patient with recurrent simultaneous multivessel coronary stent thrombosis.Oxford medical case reports · 2015Article
- A prospective multicenter study of paroxysmal nocturnal hemoglobinuria cells in patients with bone marrow failure.Cytometry. Part B, Clinical cytometry · 2014Article
- On the dynamics of neutral mutations in a mathematical model for a homogeneous stem cell population.Journal of the Royal Society, Interface · 2013Article
- Review
- Complement in organ transplantation.Current opinion in organ transplantation · 2010Review
Corrections and comments
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Authors and funding
4 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Paroxysmal nocturnal hemoglobinuria is a rare disorder of hemopoietic stem cells. Affected individuals have a triad of clinical associations - intravascular hemolysis, an increased risk of thromboembolism, and bone marrow failure. Most of the symptoms experienced in this disease occur due to the absence of complement regulatory proteins on the surface of the red blood cells. Complement activation is thus not checked and causes destruction of these cells. Eculizumab is a monoclonal antibody treatment which specifically binds to the complement protein C5, preventing its cleavage, and so halts the complement cascade and prevents the formation of the terminal complement proteins. Eculizumab prevents intravascular hemolysis, stabilizes hemoglobin levels, reduces or stops the need for blood transfusions, and improves fatigue and patient quality of life as well as reducing pulmonary hypertension, decreasing the risk of thrombosis and protecting against worsening renal function. It is not a curative therapy but has a great benefit on those with this rare debilitating condition.
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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.