Examining the Experiences of Children With Blood Disorders

• Conditions: Hemostatic Disorder; Blood Disease; Thrombotic Disorder; Venous Thromboembolism; Bleeding Disorder

• Registration Number: NCT04600609
• Lead Sponsor: University of Texas Southwestern Medical Center

Brief Summary

This is a 3-phase mixed methods study design. A literature review (Phase 1) has been completed to determine the areas of exploration and to identify challenges faced and the impact of the blood disorder on pediatric patients. Based on Phase 1, Phases 2 and 3, as proposed in this study, will be completed and will include interviews of patients diagnosed with bleeding and thrombotic disorders (phase 2). The interviews will be individual, semi-structured, and consist of open-ended questions to elicit unbiased and in-depth responses to gain an understanding of participant's perspectives on themes predetermined in the study design phase.

Detailed Description

The purpose of this study is to explore the experiences of pediatric patients with blood disorders, specifically thrombotic, including venous thromboembolism (VTE), and hemostatic or bleeding disorders. The information gleaned from this study will help identify specific challenges faced by patients with hemostatic and thrombotic disorders - information that is necessary to develop impactful, evidence-based solutions to support patients with hemostatic and thrombotic disorders cope with the physical, emotional, and psychological impact of blood disorders. Participants with blood disorders in this study will participate in open-ended, semi-structured interviews with a study investigator focusing on the psychosocial experiences of patients with diagnosis and treatment of their underlying disorder and the understanding of their diagnosis. Female participants additionally will be asked questions about the unique effects of heavy menstrual bleeding, either from their underlying blood disorder or secondary to anticoagulation, on similar aspects of the impact of having a blood disorder. Validated questionnaires will be completed to obtain concurrent quantitative data. The interviews will be recorded and transcribed. Participation is complete after the interview has finished; participants will continue to receive standard of care treatment during and beyond the study period.

Study Timeline

• Study First Submitted: 2020-09-22
• Study First Submitted QC: 2020-10-19
• Study First Posted: 2020-10-23
• Study Start: 2020-11-09
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-28
• Last Update Posted: 2025-01-31
• Primary Completion: 2026-12
• Study Completion: 2026-12

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

• Patients will be eligible to participate if they are between the ages of 12 and 21 years of age, English speaking and have a formal diagnosis of a hemostatic or thrombotic disorder. For thrombotic disorder patients, an additional eligibility criterion will be to include participants within 1 year of diagnosis.

Exclusion Criteria

• Patients will be excluded if they do not have access to the technology needed to participate in a virtual interview visit (by telephone).

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Patient confidence assessment	Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients	Patient confidence in managing blood disorder and recognizing emergencies will be assessed using a semi-structured interview with the patient
Quality of life assessment	Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients	Effect of blood disorder diagnosis on quality of life will be measured using Pediatric Quality of Life Questionnaire, Menorrhagia Impact Questionnaire, and Godin Physical Activity Questionnaire
Quality of care assessment	Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients	Impressions of quality of care received in a specialized bleeding disorders and thrombosis clinic will be assessed using a semi-structured interview with the patient
Implications on the future for the patient with the bleeding disorder	Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients	Patient's reaction to the diagnosis and its impact on their future will be assessed using a semi-structured interview with the patient

Secondary Outcome Measures

Name	Time	Method
Quality of life screening	Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients	Screening completed using Peds QOL questionnaire
Dalhousie Dypsnea screening for participants with clots	Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients	Screening completed using Dalhousie Dypsnea questionnaire
Depression screening	Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients	Screening completed using PHQ-9 questionnaire
Menorrhagia screening for participants with bleeding disorders	Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients	Screening completed using Menorrhagia impact questionnaire
Physical activity screening	Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients	Screening completed using Godin Physical Activity questionnaire

Trial Locations

Locations (1)

The University of Texas Southwestern Medical Center; 🇺🇸 Dallas, Texas, United States