Low von Willebrand factor in pediatric patients: Retrospective analysis of 293 cases informs diagnostic and therapeutic decision making.

Low von Willebrand factor (VWF) poses diagnostic and therapeutic challenges for predicting bleed risk and need for empiric hemostatic therapy, particularly in children. Retrospective review identified 293 low VWF pediatric patients and investigated clinical and laboratory features. Low activity to antigen ratio was observed in 142 patients. When evaluation included VWF activity with gain-of-function glycoprotein Ib fragments (VWF:GPIbM) assay or VWF exon 28 sequencing, low VW was excluded in the majority (62%) of these patients. Application of a condensed bleeding assessment tool to quantify bleeding symptoms and use of the VWF:GPIbM assay may reduce the number of patients with bleed risk anxiety and unnecessary hemostatic management plans.

Center-Based Quality Initiative Targets Youth Preparedness for Medical Independence: HEMO-Milestones Tool in a Comprehensive Hemophilia Clinic Setting.

BACKGROUND: Patient transition readiness self-assessment tools and investigation into patient and parent perceptions of the transition process from pediatric to adult care models have informed recognition of gaps in care, particularly for those with chronic disease. Implementation of a longitudinal transition process with patient tracking provides the opportunity for individualized education and skill building and fosters a patient-centered model. PROCEDURE: Quality improvement intervention was used to assess and improve our transition process at annual comprehensive clinic visits for patients with bleeding disorders at our tertiary care pediatric center. RESULTS: Thirty-one patients with rare bleeding disorders received an introduction to the transition process using the HEMO-milestones tool in our hemophilia comprehensive clinic from September to December 2014. The percentage of patients with documented, age-specific hemophilia knowledge/skill assessment increased from 21% to 97%. The percentage of patients with documented skill building or adult care transfer plan increased from 55% to 93%. Designated postclinic team debriefings facilitated the creation of collaborative documentation summarizing each patient's transition readiness and plan for continued skill building. CONCLUSIONS: The HEMO-milestones tool promotes a standardized approach to evaluation of self-management competency. When combined with a collaborative multidisciplinary effort, it increases plans for skill building in patients with hemophilia. This tool is easily modifiable for alternate subspecialty use and does not require extensive training for implementation.