Updated guidance for efficacy and safety outcomes for clinical trials in venous thromboembolism in children: communication from the ISTH SSC Subcommittee on Pediatric and Neonatal Thrombosis and Hemostasis

Despite the growing number of pediatric antithrombotic clinical trials, standardized safety and efficacy outcome definitions for pediatric venous thromboembolism (VTE) clinical trials have not been updated since 2011. Many recent trials have adapted the recommended definitions, leading to heterogeneity in outcomes and limiting our ability to compare studies. The International Society on Thrombosis and Haemostasis Scientific and Standardization Subcommittee (SSC) on Pediatric and Neonatal Thrombosis and Hemostasis organized a Task Force to update the efficacy and safety outcome definitions for pediatric VTE clinical trials. The outcome definitions used in the recent pediatric antithrombotic trials, definitions recommended for adult studies, and regulatory guidelines were summarized and reviewed by the Task Force as the basis for this updated guidance. Major updates to the efficacy outcomes include the removal of VTErelated mortality as a part of a composite primary outcome and explicit inclusion of all deep venous anatomic sites. Safety outcomes were updated to include a new bleeding

Automated summary

Despite the increase in pediatric antithrombotic clinical trials, the standardized outcome definitions for pediatric VTE clinical trials have not been updated since 2011. The International Society on Thrombosis and Haemostasis organized a Task Force to update the outcome definitions based on recent trials and existing guidelines. Major updates include changes in efficacy outcomes and the inclusion of new bleeding assessments in safety outcomes.