ArticleBlood2021
Cost effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura.
Article in Blood, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 46 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
46 citing papers in PubMed, 1 synthesis or guideline pooled it, 80 citations in OpenAlex.
- Adding caplacizumab to standard of care in thrombotic thrombocytopenic purpura: a systematic review and meta-analysis.Blood advances · 2023Pooled it
- Korean expert consensus on the management of thrombotic thrombocytopenic purpura.The Korean journal of internal medicine · 2026Article
- N-Acetylcysteine Therapy in Thrombotic Thrombocytopenic Purpura: A Systematic Review and Critical Appraisal.Journal of clinical medicine · 2026Review
- The complement factor B inhibitor iptacopan for relapsed and refractory immune thrombotic thrombocytopenic purpura.Thrombosis journal · 2026Article
- Presentation of HIV-Associated Thrombotic Microangiopathy and Response to Therapeutic Plasma Exchange: A 10-year Retrospective Single-Centre Cohort Study.Health science reports · 2026Article
- Thrombotic thrombocytopenic purpura in pregnancy: Lessons from a case series of three patients.The Journal of international medical research · 2026Article
- The impact of caplacizumab in the treatment of immune thrombotic thrombocytopenic purpura. A retrospective monocentric cohort study.Frontiers in pharmacology · 2026Article
- Systematic Literature Review of the Economic and Quality of Life Burden of Congenital and Immune-Mediated Thrombotic Thrombocytopenic Purpura.Journal of blood medicine · 2026Review
- Thrombocytopenia: from physiological homeostasis imbalance to mechanism-driven precision diagnosis and therapy.Frontiers in immunology · 2026Review
- Cost-effectiveness of perioperative durvalumab plus FLOT for resectable gastric and gastroesophageal junction adenocarcinoma in the United States.Frontiers in immunology · 2026Article
- Anti-ADAMTS13 Antibodies Trajectory is Associated With ADAMTS13 Recovery in Immune-Mediated TTP.American journal of hematology · 2025Article
- Cost-Effectiveness and Value of Information Analyses of Caplacizumab for the Treatment of Thrombotic Thrombocytopenic Purpura in Brazil.Applied health economics and health policy · 2025Article
- Preventative and therapeutic effects of a novel humanized anti-glycoprotein Ibα fragment of antigen-binding region in a murine model of thrombotic thrombocytopenic purpura.Journal of thrombosis and haemostasis : JTH · 2025Article
- A Descriptive 5-Year Analysis of the Demographics and Therapies for Patients With Immune Thrombotic Thrombocytopenic Purpura in the USA: A Multicenter Study of 390 Disease Episodes From 2017 to 2021.Journal of clinical apheresis · 2025Article
- Cost-effectiveness of caplacizumab in immune thrombotic thrombocytopenic purpura in the United States.Journal of managed care & specialty pharmacy · 2025Article
- Toward a Restrictive Use of Plasma Exchanges in Thrombotic Microangiopathies.Journal of the American Society of Nephrology : JASN · 2025Article
- Cost-effectiveness of sequential treatment strategies involving first-line pembrolizumab with trastuzumab and chemotherapy for unresectable metastatic HER2-positive gastric or gastroesophageal junction adenocarcinoma.Therapeutic advances in medical oncology · 2025Article
- The TMA team and TTP pathway improved outcomes in a cohort with Thrombotic thrombocytopenic purpura.PloS one · 2025Article
- Initial US tertiary health care system experience using caplacizumab in patients with immune thrombotic thrombocytopenic purpura.Annals of hematology · 2024Article
- Tailoring caplacizumab administration using ADAMTS13 activity for immune-mediated thrombotic thrombocytopenic purpura.Blood vessels, thrombosis & hemostasis · 2024Article
Corrections and comments
- Commented on by
Authors and funding
6 authors at 3 institutions in 1 country.
Funding
Abstract
Acquired thrombotic thrombocytopenic purpura (TTP) is a life-threatening disease characterized by thrombotic microangiopathy leading to end-organ damage. The standard of care (SOC) treatment is therapeutic plasma exchange (TPE) alongside immunomodulation with steroids, with increasing use of rituximab ± other immunomodulatory agents. The addition of caplacizumab, a nanobody targeting von Willebrand factor, was shown to accelerate platelet count recovery and reduce TPE treatments and hospital length of stay in TTP patients treated in 2 major randomized clinical trials. The addition of caplacizumab to SOC also led to increased bleeding from transient reductions in von Willebrand factor and increased relapse rates. Using data from the 2 clinical trials of caplacizumab, we performed the first-ever cost-effectiveness analysis in TTP. Over a 5-year period, the projected incremental cost-effectiveness ratio (ICER) in our Markov model was $1 482 260, significantly above the accepted 2019 US willingness-to-pay threshold of $195 300. One-way sensitivity analyses showed the utility of the well state and the cost of caplacizumab to have the largest effects on ICER, with a reduction in caplacizumab cost demonstrating the single greatest impact on lowering the ICER. In a probabilistic sensitivity analysis, SOC was favored over caplacizumab in 100% of 10 000 iterations. Our data indicate that the addition of caplacizumab to SOC in treatment of acquired TTP is not cost effective because of the high cost of the medication and its failure to improve relapse rates. The potential impact of caplacizumab on health system cost using longer term follow-up data merits further study.
Indexed as
Identifiers
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.