ArticleResearch and practice in thrombosis and haemostasis2024
Caplacizumab improves clinical outcomes and is well tolerated across clinically relevant subgroups of patients with immune-mediated thrombotic thrombocytopenic purpura.
Article in Research and practice in thrombosis and haemostasis, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02553317 (A Phase III Double-blind, Randomized, Parallel Group, Multicenter Placebo-controlled Trial to Study the Efficacy and Safety of Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura), which is not on this map. Cited by 3 papers.
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The trial behind it
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A Phase III Double-blind, Randomized, Parallel Group, Multicenter Placebo-controlled Trial to Study the Efficacy and Safety of Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura
Who cites it
3 citing papers in PubMed.
- Hematologic safety of caplacizumab in immune-mediated thrombotic thrombocytopenic purpura: insights from platelet-related signal detection in the FAERS database.Thrombosis journal · 2026Article
- Predicting Outcomes and Optimizing Therapy in Thrombotic Thrombocytopenic Purpura: Insights on Caplacizumab Use from a Romanian Hematology Center.Journal of clinical medicine · 2025Article
- Caplacizumab treatment in elderly patients with iTTP: Experience from the Spanish TTP Registry.HemaSphere · 2025Article
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Authors and funding
11 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Background: Immune-mediated thrombotic thrombocytopenic purpura (iTTP) may lead to microvascular thrombosis and mortality, despite patients receiving appropriate standard of care treatment (immunosuppressive therapy and therapeutic plasma exchange). Caplacizumab directly inhibits von Willebrand factor-platelet interaction and consequently prevents microthrombi formation. Objectives: This study aimed to determine the efficacy and safety of caplacizumab in diverse, clinically relevant patient subgroups. Methods: In this post hoc analysis of phase 3 HERCULES study (NCT02553317), patients were categorized by clinically relevant subgroups (prior iTTP history, iTTP severity at presentation, and initial immunosuppression regimen). Results: In patients with previous acute iTTP episodes, less severe disease at presentation, or those who received a corticosteroid-only initial immunosuppression regimen, time to platelet count response was shorter with caplacizumab vs placebo. Across all subgroups, fewer patients experienced a composite outcome of iTTP-related death, exacerbation, or major thromboembolic event on caplacizumab vs placebo. Placebo-treated patients remained at risk of exacerbations and refractoriness on either initial immunosuppression regimen (ie, corticosteroids only or corticosteroids plus rituximab). In the corticosteroids plus rituximab group, no exacerbations were reported in caplacizumab-treated patients, but 8 of the 16 (50%) patients experienced exacerbations in the placebo group. Safety outcomes were consistent with the findings of the main HERCULES study. Conclusion: Caplacizumab treatment of acute iTTP, in combination with therapeutic plasma exchange and immunosuppression, was safe and effective regardless of prior iTTP history, severity, or initial immunosuppression regimen and improved patient outcomes across clinically diverse subgroups. These findings emphasize the need for treatments with rapid onset of action that can reduce mortality and iTTP-related complications.
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