ReviewClinical and translational science2011
Paroxysmal nocturnal hemoglobinuria from bench to bedside.
Review in Clinical and translational science, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 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
30 citing papers in PubMed, 81 citations in OpenAlex.
- Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria.British journal of haematology · 2013Trial
- Article
- The incidence of paroxysmal nocturnal hemoglobinuria cell clones in the Nordic countries.Annals of hematology · 2025Article
- [Correlation between complement deposition levels on blood cells and clinical biomarkers in patients with paroxysmal nocturnal hemoglobinuria].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2025Article
- Warren Alpert Medical School of Brown University: Clinicopathologic Conference: September 20th, 2024. A Woman in her 20s with Abdominal Pain, Anemia and Thrombocytopenia.Journal of Brown hospital medicine · 2025Article
- Direct Oral Anticoagulants and Paroxysmal Nocturnal Hemoglobinuria: A Systematic Review and Update on Evidence.Cureus · 2024Review
- Article
- [Analysis and summary of clinical characteristics of 289 patients with paroxysmal nocturnal hemoglobinuria in Zhejiang Province].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2024Article
- Swiss Survey on current practices and opinions on clinical constellations triggering the search for PNH clones.Frontiers in medicine · 2023Article
- Expanding the Phenotypic Spectrum of GPI Anchoring Deficiency Due to Biallelic Variants inNeurology. Genetics · 2021Article
- Article
- MRI as a Aiagnostic Tool for Paroxysmal Nocturnal Hemoglobinuria: A Case Report.Sisli Etfal Hastanesi tip bulteni · 2021Article
- Article
- Presence of paroxysmal nocturnal hemoglobinuria in patients with idiopathic portal vein thrombosis: a single-center studyTurkish journal of medical sciences · 2020Article
- [Evaluation of the efficacy and safety of iron therapy in patients with paroxysmal nocturnal hemoglobinuria complicated with iron deficiency anemia].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2020Article
- A Rare Case of Paroxysmal Nocturnal Hemoglobinuria With Bilateral Renal Vein Thrombosis.Cureus · 2020Article
- Abdominal Surgical Emergencies in Patients with Hematological Disorders: An Exacting Experience for Surgeons.Cureus · 2019Article
- The effect of splenectomy on complement regulatory proteins in erythrocytes in β-thalassemia major.Archives of medical science : AMS · 2019Article
- Abdominal pain in combination with an unexplained hemolytic anemia are crucial signs to test for paroxysmal nocturnal hemoglobinuria: A case report.Clinical case reports · 2019Article
- Diagnostic and Treatment Challenges of Paroxysmal Nocturnal Hemoglobinuria in Uganda.Case reports in hematology · 2019Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 2 institutions in 1 country.
Funding
Abstract
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disease that presents with protean manifestations. Clinical and laboratory investigation over the past 25 years has uncovered most of the basic science underpinnings of PNH and has led to the development of a highly effective targeted therapy. PNH originates from a multipotent hematopoietic stem cell (HSC) that acquires a somatic mutation in a gene called phosphatidylinositol glycan anchor biosynthesis, class A (PIG-A). The PIG-A gene is required for the first step in glycosylphosphatidylinositol (GPI) anchor biosynthesis. Failure to synthesize GPI anchors leads to an absence of all proteins that utilize GPI to attach to the plasma membrane. Two GPI-anchor proteins, CD55 and CD59, are complement regulatory proteins; their absence on the surface of PNH cells leads to complement-mediated hemolysis. The release of free hemoglobin leads to scavenging of nitric oxide and contributes to many clinical manifestations, including esophageal spasm, fatigue, and possibly thrombosis. Aerolysin is a pore-forming toxin that binds GPI-anchored proteins and kills normal cells, but not PNH cells. A fluorescinated aerolysin variant (FLAER) binds GPI-anchor and serves as a novel reagent diagnosing PNH. Eculizumab, a humanized monoclonal antibody against C5, is the first effective drug therapy for PNH.
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.