Von Willebrand Disease: Gaining a global perspective.

INTRODUCTION: Recent guidelines for von Willebrand Disease (VWD) highlighted the challenges in diagnosis and management. Identifying the number of persons with VWD (PwVWD) internationally will help target support to aid diagnosis of PwVWD. AIM: To examine international registration rates of PwVWD, the influence of income status, geographical region and the age and sex profile. Cumulatively, these data will be used to inform future strategy from the World Federation of Haemophilia (WFH) to address unmet clinical and research needs. METHODS: Data from the 2018/2019 WFH Annual Global Survey (AGS) were analysed, providing a global perspective on VWD registration. RESULTS: Registration rates are lowest in South Asia (0.6/million population) and highest in Europe/Central Asia (50.9/million population, 0.005%), but below the expected prevalence rate (0.1%). National economic status impacted VWD registration rates, reflecting variation in access to optimal healthcare infrastructure. Females represented the majority of PwVWD globally, however, in low-income countries (LIC) males predominated. Age profile varied, with markedly higher rates of paediatric registrations in North America, Middle East and North Africa and South Asia. Rates of type 3 VWD registrations were significantly influenced by economic status (81% of VWD diagnoses in LIC), suggesting only the most severe VWD types are diagnosed in resource limited settings. CONCLUSION: Significant variation in registration rates of PwVWD exist internationally and is influenced by income status and the presence of HTC networks. Improved understanding of registration rates will enable targeting of advocacy to improve awareness, diagnosis and support for PwVWD internationally. KEY POINTS: Registration rates of People with Von Willebrand Disease (PwVWD) vary internationally and are influenced by national income status Although females represent the majority of PwVWD globally, in low income countries (LIC) males predominated, possibly related to stigma surrounding gynaecological bleeding. Rates of type 3 VWD registration were significantly influenced by economic status (81% of VWD diagnoses in LIC), suggesting only the most severe VWD types are diagnosed in resource limited settings.

Pediatric Readiness in the Emergency Department and Its Association With Patient Outcomes in Critical Care: A Prospective Cohort Study.

OBJECTIVES: Pediatric mortality in Latvia remains one of the highest among Europe. The purpose of this study was to assess the quality of pediatric acute care and pediatric readiness and determine their association with patient outcomes using a patient registry. DESIGN: This was a prospective cohort study. Pediatric readiness was measured using the weighted pediatric readiness score based on a 100-point scale. The processes of care were measured using in situ simulations to generate a composite quality score. Clinical outcome data-including PICU and hospital length of stay as well as 6-month mortality-were collected from the Pediatric Intensive Care Audit Network registry. The associations between composite quality score and weighted pediatric readiness score on patient outcomes were explored with mixed-effects regressions. SETTING: This study was conducted in all Latvian Emergency Departments and in the national PICU. PATIENTS: All patients who were transferred into the national PICU were included. INTERVENTIONS: None. MEASUREMENTS AND MAIN RESULTS: All (16/16) Latvian Emergency Departments participated with a mean composite quality score of 35.3 of 100 and a median weighted pediatric readiness score of 31 of 100. A total of 254 patients were included in the study and followed up for a mean of 436 days, of which nine died (3.5%). Higher weighted pediatric readiness score was associated significantly with lower length of stay in both the PICU and hospital (adjusted ß, -0.06; p = 0.021 and -0.36; p = 0.011, respectively) and lower 6-month mortality (adjusted odds ratio, 0.93; 95% CI, 0.88-0.98). CONCLUSIONS: These data provide a national assessment of pediatric emergency care in a European country. Pediatric readiness in the emergency department was associated with patient outcomes in this population of pediatric patients transferred to the national PICU.