ArticleAnnals of hematology2023
Brazilian experience with caplacizumab in acquired thrombotic thrombocytopenic purpura: outcomes of the expanded access program.
Article in Annals of hematology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.
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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.
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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.
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Who cites it
5 citing papers in PubMed, 8 citations in OpenAlex.
- The complement factor B inhibitor iptacopan for relapsed and refractory immune thrombotic thrombocytopenic purpura.Thrombosis journal · 2026Article
- Article
- Association between endothelial activation and stress index and all-cause mortality risk in sepsis patients: a retrospective cohort analysis utilizing the MIMIC-IV database.BMC infectious diseases · 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
- Cost-effectiveness of caplacizumab in immune thrombotic thrombocytopenic purpura in the United States.Journal of managed care & specialty pharmacy · 2025Article
Corrections and comments
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Authors and funding
4 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare disease with an acute and severe clinical presentation. The anti-von Willebrand factor caplacizumab was licensed for adults with aTTP based on prospective controlled trials. However, until now, there was no Brazilian experience with this new treatment modality. This retrospective, multicenter, single-arm, expanded access program (EAP) with caplacizumab, plasma exchange (PEX), and immunosuppression was conducted between 02/24/21 and 04/14/21, and enrolled 5 Brazilian patients with aTTP. EAP allowed access to caplacizumab in Brazil and real-world data was collected, at a time when the medication was not commercially available in Brazil. The median age was 31 years old, most patients were women (80%), and neurological manifestation was observed in 80% of cases. The median of laboratory tests was hemoglobin (Hb) of 11 g/dL, platelets (16.1 × 10
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Registered trials
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