NCT07560007

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.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1,000

participants targeted

Target at P75+ for all trials

Timeline
14mo left

Started Oct 2025

Geographic Reach
1 country

1 active site

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress33%
Oct 2025Jul 2027

Study Start

First participant enrolled

October 9, 2025

Completed
6 months until next milestone

First Submitted

Initial submission to the registry

April 16, 2026

Completed
14 days until next milestone

First Posted

Study publicly available on registry

April 30, 2026

Completed
2 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 5, 2026

Expected
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

July 5, 2027

Last Updated

April 30, 2026

Status Verified

April 1, 2026

Enrollment Period

9 months

First QC Date

April 16, 2026

Last Update Submit

April 23, 2026

Conditions

Bleeding DisorderVon Willebrand Disease (VWD)

Keywords

ISTH-BATBleeding Disorderbleeding assessmentpediatric bleeding

Outcome Measures

Primary Outcomes (13)

  • 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.

    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

Study Arms (1)

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.

Eligibility Criteria

Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

\- 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.

You may qualify if:

  • 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

You may not qualify if:

  • Lack of BAT+ on new patient visit in outpatient setting to rule out mild

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Related Publications (9)

  • 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.

    PMID: 22460356BACKGROUND

  • 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.

    PMID: 10494765BACKGROUND

  • 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.

    PMID: 16304414BACKGROUND

  • 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.

    PMID: 7794090BACKGROUND

  • 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.

    PMID: 17635722BACKGROUND

  • 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.

    PMID: 30900820BACKGROUND

  • 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.

    PMID: 19496919BACKGROUND

  • 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.

    PMID: 22974079BACKGROUND

  • 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.

    PMID: 20626619BACKGROUND

MeSH Terms

Conditions

Hemostatic Disordersvon Willebrand Diseases

Condition Hierarchy (Ancestors)

Vascular DiseasesCardiovascular DiseasesHemorrhagic DisordersHematologic DiseasesHemic and Lymphatic DiseasesBlood Coagulation Disorders, InheritedBlood Coagulation DisordersCoagulation Protein DisordersBlood Platelet DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 16, 2026

First Posted

April 30, 2026

Study Start

October 9, 2025

Primary Completion (Estimated)

July 5, 2026

Study Completion (Estimated)

July 5, 2027

Last Updated

April 30, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will not share

Locations

US(1)