ArticleJournal of managed care & specialty pharmacy2025
Cost-effectiveness of caplacizumab in immune thrombotic thrombocytopenic purpura in the United States.
Article in Journal of managed care & specialty pharmacy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 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
2 citing papers in PubMed.
- N-Acetylcysteine Therapy in Thrombotic Thrombocytopenic Purpura: A Systematic Review and Critical Appraisal.Journal of clinical medicine · 2026Review
- Systematic Literature Review of the Economic and Quality of Life Burden of Congenital and Immune-Mediated Thrombotic Thrombocytopenic Purpura.Journal of blood medicine · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
Abstract
backgroundImmune thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening thrombotic microangiopathy. Caplacizumab is the only treatment approved by the European Medicines Agency and the US Food and Drug Administration for iTTP, to be given in combination with plasma exchange therapy (PEX) and immunosuppression (IS). The National Institute for Health and Care Excellence's independent appraisal committee assessed the cost-effectiveness of caplacizumab and concluded that the addition of caplacizumab to PEX+IS is cost-effective under a patient access scheme in the United Kingdom.
objectiveTo assess the cost-effectiveness of caplacizumab in iTTP from the US payer perspective.
methodsThe National Institute for Health and Care Excellence's model was adapted to the US setting using US costs and discount rates. In contrast to previous cost-effectiveness analyses that accounted only for acute outcomes, our model consisted of a 3-month decision tree for an acute iTTP episode, followed by a Markov model to project long-term costs and outcomes (time horizon: up to 55 years; 3-monthly cycles).
resultsPatients taking caplacizumab with PEX+IS experienced an incremental gain of 2.96 life years (LYs) and 1.75 quality-adjusted LYs relative to PEX+IS alone, at an increased lifetime cost of $256,000. The incremental cost-effectiveness ratio was $86,400 per LY and $146,300 per quality-adjusted LY gained.
conclusionsConsidering willingness-to-pay thresholds of $150,000 to $200,000, the addition of caplacizumab to PEX+IS may be cost-effective compared with PEX+IS alone for the treatment of iTTP in a US setting.
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Registered trials
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