ReviewJournal of managed care & specialty pharmacy2020
Paroxysmal nocturnal hemoglobinuria: patient journey and burden of disease.
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 15 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
15 citing papers in PubMed, 1 synthesis or guideline pooled it, 27 citations in OpenAlex.
- Global Epidemiology of Paroxysmal Nocturnal Hemoglobinuria: A Systematic Literature Review.Journal of epidemiology and global health · 2026Pooled it
- Diagnostic delay and clinical outcomes of paroxysmal nocturnal hemoglobinuria: a retrospective cohort study using Japanese claims data.International journal of hematology · 2026Article
- Iptacopan monotherapy resulted in increased hemoglobin level in patients with PNH and hemoglobin ≥10 g/dL after anti-C5 therapy.HemaSphere · 2026Article
- Characterizing clinically significant extravascular hemolysis in adults with PNH on ravulizumab or eculizumab treatment.Blood advances · 2025Article
- Treatment burden in patients with paroxysmal nocturnal hemoglobinuria: an in-depth interview survey.Annals of hematology · 2025Article
- Examining Consistency Across NICE Single Technology Appraisals: A Review of Appraisals for Paroxysmal Nocturnal Haemoglobinuria.PharmacoEconomics · 2025Review
- A machine learning algorithm for the detection of paroxysmal nocturnal haemoglobinuria (PNH) in UK primary care electronic health records.Orphanet journal of rare diseases · 2024Article
- Health-related quality of life and influencing factors of patients with paroxysmal nocturnal hemoglobinuria in China.Orphanet journal of rare diseases · 2024Article
- Mapping the EORTC QLQ-C30 onto the EQ-5D-5L index for patients with paroxysmal nocturnal hemoglobinuria in France.Journal of comparative effectiveness research · 2023Article
- Paroxysmal Nocturnal Hemoglobinuria: Current Management, Unmet Needs, and Recommendations.Journal of blood medicine · 2023Review
- Article
- Review
- 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
- The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient survey.Annals of hematology · 2022Article
- Managing Fatigue in Patients with Paroxysmal Nocturnal Hemoglobinuria: A Patient-Focused Perspective.Journal of blood medicine · 2022Review
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
Patients with paroxysmal nocturnal hemoglobinuria (PNH) often experience a lengthy path to diagnosis. Fewer than 40% of patients with PNH receive a diagnosis within 12 months of symptom onset, and 24% of all PNH diagnoses can take 5 years or longer. Diagnostic delay is a source of distress and can affect emotional well-being for patients with PNH. In PNH disease management, patients and care providers focus on risk of organ failure and mortality related to disease progression; nonetheless, patients' health-related quality of life (HRQOL) is largely affected by extensive treatment requirements and nonfatal complications of disease, such as fatigue. In particular, thrombosis is associated with significant impairments in physical and social functioning and global health status and significant fatigue. Among patients with anemia who are transfusion dependent, the burden of transfusion is considerable. Transfusion dependence has a negative effect on HRQOL; is associated with risks and complications, including iron overload; and results in lost productivity due to travel times to and time spent at infusion centers.
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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.