Examining the Experiences of Children With Blood Disorders

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.

Study Overview

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

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 Type: Observational
• Enrollment (Actual): 15

Contacts and Locations

Study Locations

• United States
  • Texas
    • Dallas, Texas, United States, 75235
      • The University of Texas Southwestern Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 21 years (Child, Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Study population will include children (defined as persons who have not attained the legal age for consent to treatments or procedures involved in the research, under the applicable law of the jurisdiction in which the research will be conducted), patients (defined as individuals in a clinical setting with whom there is a treatment relationship), and non-English speakers.

Patients must meet the eligibility criteria in order to participate in this trial.

Description

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 Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Quality of life assessment	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	Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients
Patient confidence assessment	Patient confidence in managing blood disorder and recognizing emergencies will be assessed using a semi-structured interview with the patient	Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients
Quality of care assessment	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	Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients
Implications on the future for the patient with the bleeding disorder	Patient's reaction to the diagnosis and its impact on their future will be assessed using a semi-structured interview with the patient	Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: University of Texas Southwestern Medical Center
• Investigators: Principal Investigator: Ayesha Zia, MD, University of Texas Southwestern Medical Center

Publications and helpful links

General Publications

• Parker M, Hannah M, Zia A. "If I wasn't a girl": Experiences of adolescent girls with heavy menstrual bleeding and inherited bleeding disorders. Res Pract Thromb Haemost. 2022 May 30;6(4):e12727. doi: 10.1002/rth2.12727. eCollection 2022 May.

Study record dates

Study Major Dates

• Study Start (Actual): November 9, 2020
• Primary Completion (Actual): June 27, 2021
• Study Completion (Actual): June 27, 2021

Study Registration Dates

• First Submitted: September 22, 2020
• First Submitted That Met QC Criteria: October 19, 2020
• First Posted (Actual): October 23, 2020

Study Record Updates

• Last Update Posted (Actual): February 5, 2026
• Last Update Submitted That Met QC Criteria: February 4, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Vascular Diseases; Cardiovascular Diseases; Embolism and Thrombosis; Hemorrhagic Disorders; Thromboembolism; Hemic and Lymphatic Diseases; Thrombosis; Hematologic Diseases; Venous Thromboembolism; Hemostatic Disorders
• Other Study ID Numbers: STU-2020-0772

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No