Clinical trial NCT04600609: Examining the Experiences of Children With Blood Disorders
Official title: Examining the Experiences of Children With Blood Disorders
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Blood Disease
Thrombotic Disorder
Venous Thromboembolism
Hemostatic Disorder
Bleeding Disorder
Lead sponsor: University of Texas Southwestern Medical Center
Sponsor class: OTHER
First posted: 2020-10-23
Enrollment: 50
Sex: ALL
Minimum age (years): 12
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
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.

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.

Eligibility criteria: 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).

Primary outcomes:
measure: Quality of life assessment
timeFrame: Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients
measure: Patient confidence assessment
timeFrame: Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients
measure: Quality of care assessment
timeFrame: Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients
measure: Implications on the future for the patient with the bleeding disorder
timeFrame: Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients
Secondary outcomes:
measure: Depression screening
timeFrame: Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients
measure: Quality of life screening
timeFrame: Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients
measure: Menorrhagia screening for participants with bleeding disorders
timeFrame: Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients
measure: Dalhousie Dypsnea screening for participants with clots
timeFrame: Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients
measure: Physical activity screening
timeFrame: Within 1 year of diagnosis for clot patients; after formal diagnosis for bleeding disorder patients
Other outcomes:
Locations:
city: Dallas
country: United States
facility: The University of Texas Southwestern Medical Center
state: Texas
Study officials:
affiliation: University of Texas Southwestern Medical Center
name: Ayesha Zia, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.