Empowering Adolescents and Young Adults With Sickle Cell Disease as Partners in Treatment Decision Making (EMPOWER-AYA)

Study Overview
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Brief Summary
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This study will evaluate the acceptability, feasibility, and preliminary efficacy of a shared decision making intervention for adolescents and young adults (AYAs) with sickle cell disease (SCD). 30 AYAs with SCD (ages 15-25 years) and their caregivers and 10 healthcare providers will participate in the single-arm pilot trial. Study procedures for this future clinical trial will be specified once intervention development, usability testing, and implementation planning phases (currently underway) have been completed.

Official Title
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Empowering Adolescents and Young Adults With Sickle Cell Disease as Partners in Treatment Decision Making (EMPOWER-AYA)

Conditions
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Sickle Cell Disease

Intervention / Treatment
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* Behavioral: Shared Decision Making Intervention


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients diagnosed with SCD ages 15-25 years receiving treatment at Nemours or ChristianaCare and their caregivers will be included. Patients must be deemed candidates for initiation of one or more disease-modifying therapies by their SCD healthcare provider. Nemours and ChristianaCare SCD healthcare providers will also be included. English language fluency is a requirement for all participants. Exclusion Criteria: Patients/ caregivers who previously participated in usability testing of the intervention will be excluded. Patients will also be excluded if their medical status or cognitive functioning preclude completion of interviews or if no legal guardian is available to provide consent (for patients < 18 years). Providers still completing training will be excluded.

Ages Eligible for Study
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Minimum Age: 15 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Supportive Care
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Shared Decision Making Intervention<br> | Behavioral: Shared Decision Making Intervention<br>* We are currently developing a shared decision making intervention for AYAs with SCD to provide decision supports for disease-modifying therapies. As the intervention is currently in development, additional details will be provided once development and usability testing phases have been completed.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Acceptability of Intervention Measure (AIM) | Questionnaire assessing healthcare provider perspectives on acceptability of the intervention. Total score ranges from 4-20, with higher scores indicating greater acceptability. | Within 2 weeks post-intervention | 
| Patient/Caregiver Satisfaction Questionnaire | Survey assessing patient/caregiver satisfaction with the intervention. Survey will be developed by the research team, and additional details will be included here once items and scoring are finalized. | Within 2 weeks post-intervention | 
| Feasibility of Intervention Measure (FIM) | Questionnaire assessing healthcare provider perspectives on the feasibility of the intervention. Total score ranges from 4-20, with higher scores indicating greater feasibility. | Within 2 weeks post-intervention | 
| Participant Recruitment Rate | Rate of participant enrollment and retention in the study | Time of enrollment through study completion, approximately 12 months | 
| Shared Decision Making Questionnaire (SDM-Q-9) | Questionnaire assessing patient- and caregiver-reported engagement in decision making. Total score ranges from 0 to 100, with higher scores indicating greater engagement in decision making. | Within 2 weeks post-intervention | 
| Disease-Modifying Therapy Knowledge Questionnaire | Questionnaire assessing patient and caregiver knowledge of disease modifying-therapies for sickle cell disease. Survey will be developed by the research team, and additional details will be included here once items and scoring are finalized. | Within 2 weeks post-intervention | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Initiation of disease-modifying therapy | Proportion of patients who agree to initiate a disease-modifying therapy following use of the shared decision making intervention | Within 6 months post-intervention |