ReviewJournal of managed care & specialty pharmacy2020
Paroxysmal nocturnal hemoglobinuria: current treatments and unmet needs.
Review in Journal of managed care & specialty pharmacy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers, 1 of them a synthesis that pooled it.
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
21 citing papers in PubMed, 1 synthesis or guideline pooled it, 37 citations in OpenAlex.
- Efficacy and Safety of the C3 Inhibitor Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria: A Systematic Review and Meta-Analysis.European journal of haematology · 2026Pooled it
- Efficacy and safety of vemircopan as monotherapy in patients with paroxysmal nocturnal hemoglobinuria.Blood advances · 2026Trial
- Patient-reported outcomes in patients with paroxysmal nocturnal hemoglobinuria treated with crovalimab and approved C5 inhibitors in the phase III COMMODORE 2 and 1 studies.Annals of hematology · 2025Trial
- Normalization of Hemoglobin, Lactate Dehydrogenase, and Fatigue in Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Pegcetacoplan.Drugs in R&D · 2024Trial
- Predictors for improvement in patient-reported outcomes: post hoc analysis of a phase 3 randomized, open-label study of eculizumab and ravulizumab in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria.Annals of hematology · 2024Trial
- Pegcetacoplan controls hemolysis in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria.Blood advances · 2023Trial
- 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
- 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
- Advancements in complement inhibition for PNH and primary complement-mediated thrombotic microangiopathy.Blood advances · 2025Review
- Article
- Beyond Recycling Antibodies: Crovalimab's Molecular Design Enables Four-Weekly Subcutaneous Injections for PNH Treatment.International journal of molecular sciences · 2024Article
- Review
- Health-related quality of life and influencing factors of patients with paroxysmal nocturnal hemoglobinuria in China.Orphanet journal of rare diseases · 2024Article
- Safety Profile of Monoclonal Antibodies and Subsequent Drug Developments in the Treatment of Paroxysmal Nocturnal Hemoglobinuria.Medicina (Kaunas, Lithuania) · 2024Review
- [Expert consensus on clonal screening and monitoring of complement inhibitor therapy in paroxysmal nocturnal hemoglobinuria (2024)].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2024Article
- Descriptive, real-world treatment patterns, resource use, and total cost of care among eculizumab- and ravulizumab-treated members with paroxysmal nocturnal hemoglobinuria.Journal of managed care & specialty pharmacy · 2023Article
- Article
- Thromboembolic events in patients with paroxysmal nocturnal hemoglobinuria (PNH): Real world data of a Greek nationwide multicenter retrospective study.Frontiers in oncology · 2023Article
- The burden of illness of patients with paroxysmal nocturnal haemoglobinuria receiving C5 inhibitors in France, Germany and the United Kingdom: Patient-reported insights on symptoms and quality of life.European journal of haematology · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The current standard of care for paroxysmal nocturnal hemoglobinuria (PNH) are the C5 inhibitors eculizumab and ravulizumab, both monoclonal antibodies designed to target the complement protein C5, thereby preventing its cleavage and the formation of the terminal attack complex. C5 inhibitors have yielded substantial improvements in the treatment of PNH and changed the mortality and morbidity, as well as health-related quality of life of patients with the disease. These treatments target underlying intravascular hemolysis; however, they do not address extravascular hemolysis, resulting in incomplete response and remaining symptoms in some patients. Therefore, despite treatment with a C5 inhibitor, some patients still experience anemia with associated fatigue, transfusion needs, and impaired health-related quality of life.
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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.