Identifying Children with HEreditary Coagulation disorders (iCHEC): a protocol for a prospective cohort study

Area of Science:

• Paediatric Haematology
• Inherited Bleeding Disorders
• Diagnostic Accuracy

Background:

• Obtaining accurate bleeding histories in children with suspected inherited bleeding disorders is challenging due to subtle symptoms.
• Existing bleeding assessment tools (BATs) lack sensitivity, efficiency, and flexibility for paediatric use.
• A novel BAT, the iCHEC (identifying Children with HEreditary Coagulation disorders) BAT, was developed to address these limitations.

Purpose of the Study:

• To evaluate the diagnostic accuracy of the iCHEC BAT as a screening tool for children suspected of having an inherited bleeding disorder.
• To assess the sensitivity, efficiency, and flexibility of the iCHEC BAT in a paediatric population.

Main Methods:

• Prospective cohort study involving children (0-18 years) presenting at tertiary haematology clinics.
• Children/guardians will complete the iCHEC BAT, featuring paediatric-specific symptoms and qualitative questions.
• Diagnostic accuracy will be evaluated using receiver operator characteristic curves against laboratory assessment as the reference standard.

Main Results:

• The iCHEC BAT incorporates paediatric-specific bleeding symptoms to increase sensitivity.
• An online, self-administered version enhances the efficiency of the questionnaire.
• Inclusion of questions on the timing of bleeding symptoms improves flexibility for developmental changes.

Conclusions:

• The iCHEC BAT demonstrates potential as an improved screening tool for inherited bleeding disorders in children.
• Further evaluation will confirm its diagnostic accuracy and clinical utility in paediatric haematology.
• The study aims to enhance the reliable identification of bleeding disorders in young patients.