Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BAT CBDI patients | -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. |
Not provided
| Measure | Description | Time Frame |
|---|---|---|
| BAT+ Classification | Dichotomous BAT classification (BAT+ vs BAT-) based on predefined age- and sex-specific BAT cutoffs. | From January 2019, through December 1, 2024 |
| Bleeding Assessment Tool (BAT) Score | Total BAT score calculated according to standardized criteria to quantify bleeding symptoms. | From January 2019, through December 1, 2024 |
| Probability of Diagnosed Bleeding Disorder Based on BAT Score | 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. | From January 2019, through December 1, 2024 |
| Probability of Diagnosed Bleeding Disorder Based on BAT+ Status | Predicted pretest probability of a confirmed bleeding disorder diagnosis estimated using logistic regression models with BAT+ classification as the primary predictor. | From January 2019, through December 1, 2024 |
| Confirmed Bleeding Disorder Diagnosis by Category | Final clinical diagnosis categorized as von Willebrand disease, platelet function disorder, coagulation factor deficiency, fibrinolytic disorder, or no bleeding disorder, using standardized diagnostic criteria. | From January 2019, through December 1, 2024 |
| Diagnostic Accuracy of BAT | 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. |
Not provided
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
- 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.
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 22460356 | Background | O'Brien SH. An update on pediatric bleeding disorders: bleeding scores, benign joint hypermobility, and platelet function testing in the evaluation of the child with bleeding symptoms. Am J Hematol. 2012 May;87 Suppl 1:S40-4. doi: 10.1002/ajh.23157. Epub 2012 Mar 28. | |
| 10494765 | Background | Castaman G, Eikenboom JC, Bertina RM, Rodeghiero F. Inconsistency of association between type 1 von Willebrand disease phenotype and genotype in families identified in an epidemiological investigation. Thromb Haemost. 1999 Sep;82(3):1065-70. |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D020141 | Hemostatic Disorders |
| D014842 | von Willebrand Diseases |
| ID | Term |
|---|---|
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D006474 | Hemorrhagic Disorders |
| D006402 | Hematologic Diseases |
Not provided
Not provided
Not provided
Not provided
Not provided
| From January 2019, through December 1, 2024 |
| Diagnostic Accuracy of BAT+ | 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. | From January 2019, through December 1, 2024 |
| Likelihood Ratios for BAT | Positive likelihood ratio for BAT at the initial cutoff score of ≥3 and at optimized cutoffs derived from ROC analysis. | From January 2019, through December 1, 2024 |
| Likelihood Ratios for BAT+ | Positive likelihood ratio for BAT+ at the initial cutoff score of ≥6 and at optimized cutoffs derived from ROC analysis. | From January 2019, through December 1, 2024 |
| Area Under the ROC Curve (AUC) for BAT | Area under the receiver operating characteristic (ROC) curve for BAT scores derived from logistic regression models discriminating confirmed bleeding disorder diagnosis (yes/no). | From January 2019, through December 1, 2024 |
| Area Under the ROC Curve (AUC) for BAT+ | Area under the ROC curve for BAT+ classification derived from logistic regression models discriminating confirmed bleeding disorder diagnosis (yes/no). | From January 2019, through December 1, 2024 |
| Optimized BAT Cutoff Score | Optimized BAT cutoff score identified using Youden's Index based on ROC curve analysis. | From January 2019, through December 1, 2024 |
| Optimized BAT+ Cutoff Score | Optimized BAT+ cutoff identified using Youden's Index based on ROC curve analysis. | From January 2019, through December 1, 2024 |
| 16304414 | Background | Hayward CP. Diagnosis and management of mild bleeding disorders. Hematology Am Soc Hematol Educ Program. 2005:423-8. doi: 10.1182/asheducation-2005.1.423. |
| 7794090 | Background | Sramek A, Eikenboom JC, Briet E, Vandenbroucke JP, Rosendaal FR. Usefulness of patient interview in bleeding disorders. Arch Intern Med. 1995 Jul 10;155(13):1409-15. |
| 17635722 | Background | Rodeghiero F, Tosetto A, Castaman G. How to estimate bleeding risk in mild bleeding disorders. J Thromb Haemost. 2007 Jul;5 Suppl 1:157-66. doi: 10.1111/j.1538-7836.2007.02520.x. |
| 30900820 | Background | Bui J, Martyres D, James PD, Grabell J, Wu J, Steele M, Silva M, Rand ML, Blanchette VS, Barrowman N, Klaassen RJ. Validation of the school age self-administered pediatric bleeding questionnaire (Self-PBQ) in children aged 8-12 years. Pediatr Blood Cancer. 2019 Jun;66(6):e27709. doi: 10.1002/pbc.27709. Epub 2019 Mar 22. |
| 19496919 | Background | Bowman M, Riddel J, Rand ML, Tosetto A, Silva M, James PD. Evaluation of the diagnostic utility for von Willebrand disease of a pediatric bleeding questionnaire. J Thromb Haemost. 2009 Aug;7(8):1418-21. doi: 10.1111/j.1538-7836.2009.03499.x. Epub 2009 May 30. No abstract available. |
| 22974079 | Background | Rydz N, James PD. The evolution and value of bleeding assessment tools. J Thromb Haemost. 2012 Nov;10(11):2223-9. doi: 10.1111/j.1538-7836.2012.04923.x. |
| 20626619 | Background | Rodeghiero F, Tosetto A, Abshire T, Arnold DM, Coller B, James P, Neunert C, Lillicrap D; ISTH/SSC joint VWF and Perinatal/Pediatric Hemostasis Subcommittees Working Group. ISTH/SSC bleeding assessment tool: a standardized questionnaire and a proposal for a new bleeding score for inherited bleeding disorders. J Thromb Haemost. 2010 Sep;8(9):2063-5. doi: 10.1111/j.1538-7836.2010.03975.x. No abstract available. |
| D006425 |
| Hemic and Lymphatic Diseases |
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D020147 | Coagulation Protein Disorders |
| D001791 | Blood Platelet Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |