ReviewCost effectiveness and resource allocation : C/E2025
Disease-modifying treatments in paroxysmal nocturnal hemoglobinuria: a systematic review of economic evaluations.
Review in Cost effectiveness and resource allocation : C/E, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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
0 citing papers in PubMed.
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Corrections and comments
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Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundParoxysmal Nocturnal Hemoglobinuria (PNH) is a rare, life-threatening hematologic disorder characterized by chronic hemolysis, bone marrow and organ failure, and thrombotic events. Without effective treatment, PNH can lead to severe complications, including organ damage, increased morbidity, and heightened mortality risk. The high expense and therapeutic revolution that has emerged in PNH necessitates a careful appraisal of its cost-effectiveness. This study aimed to systematically review the cost-effectiveness of disease-modifying therapies (DMTs) in PNH.
methodsA comprehensive search syntax was developed to identify relevant studies using online databases. Criteria used in health economic evaluations include costs, effectiveness assessment with life-years gained (LYG) or quality-adjusted life-years (QALY), incremental cost-effectiveness ratio (ICER), and other clinico-economic outcomes. To enable comparison across different countries, the results of economic evaluations allow for the comparison of costs and ICERs across all studies, which were converted into United States Dollars (USD) values using the time model developed. The quality of the studies was assessed using the Quality of Health Economic Studies (QHES) instrument. This study is conducted and reported following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) statement.
resultsNine papers were included in the 351 screened records. Pegcetacoplan was a dominant (i.e., less costly and more effective) option compared to C5 complement inhibitors and iptacopan. Eculizumab was not cost-effective compared with other DMTs or the standard of care. The current annual price of $12,300-$13,100 for adding danicopan to ravulizumab was not cost-effective compared to ravulizumab alone. The results of nine studies indicated that the majority of costs are related to the DMTs themselves.
conclusionThe cost-effectiveness analyses of DMTs in PNH suggest that the newer options, pegcetacoplan and iptacopan, are cost-effective and provide greater clinical utility than complement C5 inhibitors, especially eculizumab.
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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 Socratic graph.