ArticleHematology, transfusion and cell therapy
Consensus statement for diagnosis and treatment of paroxysmal nocturnal haemoglobinuria.
Article in Hematology, transfusion and cell therapy. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 papers, 2 of them syntheses that pooled it.
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Who cites it
35 citing papers in PubMed, 2 syntheses or guidelines pooled it, 52 citations in OpenAlex.
- Global Epidemiology of Paroxysmal Nocturnal Hemoglobinuria: A Systematic Literature Review.Journal of epidemiology and global health · 2026Pooled it
- [Guidelines for the diagnosis and management of paroxysmal nocturnal hemoglobinuria (2024)].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2024Guideline
- Haploidentical allogeneic haematopoietic stem cell transplantation for paroxysmal nocturnal haemoglobinuria: a retrospective analysis.Annals of hematology · 2026Article
- [How I diagnose and treat paroxysmal nocturnal hemoglobinuria].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2026Article
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- Paroxysmal nocturnal hemoglobinuria with a positive Coombs test presenting as acute kidney injury: a case report.Frontiers in medicine · 2026Article
- Optimizing Care in Patients with Paroxysmal Nocturnal Hemoglobinuria: Managing Suboptimal Response and Uncontrolled Disease.Journal of blood medicine · 2026Article
- Paroxysmal Nocturnal Hemoglobinuria-Driven Hepatic Vein Thrombosis: A Case of Budd-Chiari Syndrome in Disguise.Cureus · 2025Article
- [Significance of the 50% hemolytic complement in hemolysis assessment and efficacy of eculizumab in patients with paroxysmal nocturnal hemoglobinuria].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2025Article
- Paroxysmal Nocturnal Hemoglobinuria: Unraveling Its Molecular Pathogenesis and Advancing Targeted Therapeutic Strategies.Diseases (Basel, Switzerland) · 2025Review
- Clinical characteristics and management of paroxysmal nocturnal hemoglobinuria in the Middle East: a narrative review.Clinical and experimental medicine · 2025Review
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- Article
- Effect of Target-Mediated Disposition on Iptacopan Clinical Pharmacokinetics in Participants with Normal or Impaired Hepatic Function.Clinical pharmacology and therapeutics · 2025Article
- Article
- Real-World Evidence of the Disease Burden and Economic Impact of Paroxysmal Nocturnal Hemoglobinuria in Italy.Journal of clinical medicine · 2025Article
- [Limitations and challenges of glucocorticoids in the treatment of paroxysmal nocturnal hemoglobinuria].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2025Article
- Clinical characteristics and management of paroxysmal nocturnal haemoglobinuria in Latin America: a narrative review.Annals of hematology · 2025Review
- Monitoring and Treatment of Paroxysmal Nocturnal Hemoglobinuria in Patients with Aplastic Anemia in Asia: An Expert Consensus.International journal of molecular sciences · 2024Review
- Thrombosis in Paroxysmal Nocturnal Hemoglobinuria (PNH): From Pathogenesis to Treatment.International journal of molecular sciences · 2024Review
Corrections and comments
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Authors and funding
9 authors at 8 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Paroxysmal nocturnal hemoglobinuria is a chronic, multi-systemic, progressive and life-threatening disease characterized by intravascular hemolysis, thrombotic events, serious infections and bone marrow failure. Paroxysmal nocturnal hemoglobinuria results from the expansion of a clone of hematopoietic cells that due to an inactivating mutation of the X-linked gene PIG-A are deficient in glycosylphosphatidylinositol-linked proteins. Early diagnosis, using flow cytometry performed on peripheral blood, the gold standard test to confirm the diagnosis of paroxysmal nocturnal hemoglobinuria, is essential for improved patient management and prognosis. The traditional therapy for paroxysmal nocturnal hemoglobinuria includes blood transfusion, anti-thrombosis prophylaxis or allogeneic bone marrow transplantation. The treatment that has recently become available is the complement blockade by the anti-C5 monoclonal antibody eculizumab. In this consensus, we are aiming to review the diagnosis and treatment of the paroxysmal nocturnal hemoglobinuria patients, as well as the early recognition of its systemic complications. These procedures express the opinions of experts and have been based on the best available evidence and international guidelines, with the purpose of increasing benefits and reducing harm to patients.
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