Publications by authors named "C Male"

Introduction: Evidence-based clinical practice guidelines drive optimal patient care and facilitate access to high-quality treatment. Creating guidelines for rare diseases such as haemophilia, where evidence does not often come from randomized controlled trials but from non-randomized and well-designed observational studies and real-world data, is challenging. The methodology used for assessing available evidence should consider this critical fact.

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Article Synopsis
  • The study analyzes data from the Kids-DOTT trial to investigate the treatment and outcomes of children with cerebral sinovenous thrombosis (CSVT) compared to those with other types of venous thromboembolism (VTE).
  • CSVT was found to be more common in neonates and young children, often linked to infections, while treatment involved varying durations of anticoagulation, with no significant difference in outcomes between 6 weeks and longer treatments.
  • The findings suggest that 6 weeks of anticoagulant therapy is safe and effective for treating acute pediatric CSVT, but caution is advised in generalizing results due to the nature of subgroup analysis.
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Article Synopsis
  • * A study involving 2,727 children showed that ICH occurred in 2.2% of cases, mostly before the age of one, with a significantly higher incidence in hemophilia A compared to hemophilia B.
  • * Early diagnosis of hemophilia is crucial, as 40% of ICH cases happened before the diagnosis, and preventive treatment could potentially lower the incidence of ICH if initiated promptly.
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Introduction: Patients with congenital heart disease (ConHD) are at increased risk for adverse cardiac events. Predicting long-term outcomes and guidance of patient management might benefit from a range of (new) biomarkers. This is a rapidly evolving field with potentially large consequences for clinical decision making.

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