Bleeding Disorder, Von Willebrand Disease (VWD)
Conditions
Keywords
ISTH-BAT, Bleeding Disorder, bleeding assessment, pediatric bleeding
Brief summary
This study looks at whether adding simple family history questions to a standard bleeding questionnaire can better identify children who truly have mild bleeding disorders. Because young children often haven't had major bleeding challenges yet, their own symptoms may not tell the full story, so family history could provide important extra information. By reviewing several years of clinic data, the researchers aim to see if this combined approach helps doctors decide more accurately who needs further testing, especially in children under 8 years old.
Detailed description
This study focuses on improving how doctors evaluate children who may have a mild bleeding disorder, such as von Willebrand disease or platelet function disorders. Doctors already use a standardized bleeding questionnaire to record a child's bleeding symptoms, but this can be difficult in younger children who often haven't had major bleeding challenges like surgery or dental work. To address this, the researchers added a small number of family history questions to the existing tool to see if knowing about bleeding problems in relatives helps predict whether a child truly has a bleeding disorder. The study reviews several years of existing medical records from children and young adults seen in a pediatric hematology clinic. By comparing the standard questionnaire with the expanded version that includes family history, the researchers want to see which approach more accurately identifies patients who go on to receive a confirmed diagnosis. The goal is to help clinicians decide more confidently who needs further testing and who does not, reducing unnecessary and costly lab work while improving diagnosis, especially for children under 8 years old, where bleeding symptoms alone may be misleading.
Interventions
None listed
Sponsors
Study design
Eligibility
Inclusion criteria
* All individuals seen at CCHMC CBDI clinic for mild bleeding disorder with completed BAT+ at their new patient visit to Cincinnati Children's Hospital Medical Center (Base Campus or Liberty Campus) since 2019. * If patient has multiple BAT+ scores will utilize the BAT+ score done at the new patient Hematology clinic CCHMC
Exclusion criteria
* Lack of BAT+ on new patient visit in outpatient setting to rule out mild
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| BAT+ Classification | From January 2019, through December 1, 2024 | Dichotomous BAT classification (BAT+ vs BAT-) based on predefined age- and sex-specific BAT cutoffs. |
| Bleeding Assessment Tool (BAT) Score | From January 2019, through December 1, 2024 | Total BAT score calculated according to standardized criteria to quantify bleeding symptoms. |
| Probability of Diagnosed Bleeding Disorder Based on BAT Score | From January 2019, through December 1, 2024 | Predicted pretest probability of a confirmed bleeding disorder diagnosis (vWD, platelet function disorder, coagulation factor deficiency, or fibrinolytic disorder) estimated using logistic regression models with BAT score as a continuous predictor. |
| Probability of Diagnosed Bleeding Disorder Based on BAT+ Status | From January 2019, through December 1, 2024 | Predicted pretest probability of a confirmed bleeding disorder diagnosis estimated using logistic regression models with BAT+ classification as the primary predictor. |
| Confirmed Bleeding Disorder Diagnosis by Category | From January 2019, through December 1, 2024 | Final clinical diagnosis categorized as von Willebrand disease, platelet function disorder, coagulation factor deficiency, fibrinolytic disorder, or no bleeding disorder, using standardized diagnostic criteria. |
| Diagnostic Accuracy of BAT | From January 2019, through December 1, 2024 | Diagnostic accuracy of the Bleeding Assessment Tool (BAT) for identifying a confirmed bleeding disorder, including sensitivity, specificity, positive predictive value (PPV), and negative predictive value (NPV), using an initial cutoff score of ≥3. Optimized cutoff scores will be reassessed using ROC analysis and Youden's Index. |
| Diagnostic Accuracy of BAT+ | From January 2019, through December 1, 2024 | Diagnostic accuracy of the BAT+ classification for identifying a confirmed bleeding disorder, including sensitivity, specificity, PPV, and NPV, using an initial cutoff score of ≥6. Optimized cutoffs will be reassessed using ROC analysis and Youden's Index. |
| Likelihood Ratios for BAT | From January 2019, through December 1, 2024 | Positive likelihood ratio for BAT at the initial cutoff score of ≥3 and at optimized cutoffs derived from ROC analysis. |
| Likelihood Ratios for BAT+ | From January 2019, through December 1, 2024 | Positive likelihood ratio for BAT+ at the initial cutoff score of ≥6 and at optimized cutoffs derived from ROC analysis. |
| Area Under the ROC Curve (AUC) for BAT | From January 2019, through December 1, 2024 | Area under the receiver operating characteristic (ROC) curve for BAT scores derived from logistic regression models discriminating confirmed bleeding disorder diagnosis (yes/no). |
| Area Under the ROC Curve (AUC) for BAT+ | From January 2019, through December 1, 2024 | Area under the ROC curve for BAT+ classification derived from logistic regression models discriminating confirmed bleeding disorder diagnosis (yes/no). |
| Optimized BAT Cutoff Score | From January 2019, through December 1, 2024 | Optimized BAT cutoff score identified using Youden's Index based on ROC curve analysis. |
| Optimized BAT+ Cutoff Score | From January 2019, through December 1, 2024 | Optimized BAT+ cutoff identified using Youden's Index based on ROC curve analysis. |
Countries
United States