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von Willebrand factor antigen--a possible biomarker of disease activity in childhood central nervous system
Tania Cellucci1, Pascal N Tyrrell, Eleanor Pullenayegum
1Division of Rheumatology, The Hospital for Sick Children, 555 University Avenue, Toronto, Ontario M5G 1X8, Canada.
Insights
Von Willebrand factor (vWF) antigen levels decreased after treatment in children with childhood primary CNS vasculitis (cPACNS), potentially serving as a biomarker for disease activity and treatment response, unlike CRP and ESR. Further controlled studies are needed.
Area of Science:
- Pediatric Neurology
- Immunology
- Vascular Biology
Background:
- Childhood primary CNS vasculitis (cPACNS) is a rare condition affecting brain blood vessels.
- Monitoring disease activity and treatment response in cPACNS can be challenging.
- Current inflammatory markers like CRP and ESR may not consistently reflect disease status.
Purpose of the Study:
- To establish a trajectory for von Willebrand factor (vWF) antigen levels post-treatment in pediatric cPACNS.
- To compare vWF antigen changes with disease activity and other inflammatory markers.
- To assess the potential of vWF antigen as a biomarker in cPACNS.
Main Methods:
- A single-center cohort study included 39 children diagnosed with cPACNS.
- Data collected included demographics, clinical presentation, laboratory results, imaging, and histology.
- Outcome measures involved physician global assessment of disease activity and serial vWF antigen measurements.
Main Results:
- Neurological deficits and disease activity significantly improved during the 18-month follow-up.
- vWF antigen levels were elevated at diagnosis in 65% of patients and decreased significantly post-treatment.
- Unlike fluctuating CRP and ESR, vWF antigen levels showed a significant decrease, and higher initial levels correlated with better disease activity at 12 months.
Conclusions:
- Children with cPACNS demonstrated improvement over time.
- vWF antigen shows promise as a biomarker for monitoring cPACNS disease activity and predicting treatment response.
- Further controlled studies are required to validate the sensitivity and specificity of vWF antigen in cPACNS.
Objective:
The main objective was to develop a trajectory for von Willebrand factor (vWF) antigen in childhood primary CNS vasculitis (cPACNS) after treatment and compare this with disease activity and other inflammatory markers.
Methods:
A single-centre cohort study of consecutive children diagnosed with cPACNS was performed. Demographic, clinical, laboratory, imaging and histological data were collected at diagnosis and during standardized clinic visits. Outcome measures included disease activity measured by physician global assessment and serial measures of vWF antigen. Analysis included descriptive statistics and parametric methods.
Results:
The study cohort consisted of 39 children diagnosed with cPACNS: 25 with angiography-negative cPACNS and 14 with angiography-positive disease. Twenty-one patients were female, median age at diagnosis was 9.8 years, and median follow-up was 18 months. All patients presented with neurological deficits. Disease activity and neurological outcome improved significantly during follow-up. vWF antigen levels were increased at diagnosis in 65% of children with cPACNS and were decreased significantly after treatment. In contrast, levels of CRP and ESR fluctuated over time. Higher vWF antigen levels at diagnosis were associated with lower measures of disease activity at 12 months.
Conclusion:
In our study, all children with cPACNS improved over time. Changes in CRP and ESR, other laboratory tests, and MRI did not consistently reflect altered disease activity. However, vWF antigen may help clinicians to identify changes in disease activity during follow-up and predict treatment response. Controlled studies are necessary to evaluate the sensitivity and specificity of vWF antigen as a biomarker of disease activity.
