Empowering Adolescents and Young Adults with Sickle Cell Disease: Development of a Shared Decision-Making Toolkit for Disease-Modifying Therapies

Introduction: While hydroxyurea remains the mainstay therapy for sickle cell disease (SCD), the treatment landscape is evolving rapidly, with three additional drugs now approved to prevent or treat SCD complications (Ballas, 2020). Individuals with SCD and clinicians face decisions about multiple disease-modifying therapies, each with unique clinical indications and tradeoffs impacting quality of life (Ballas, 2020; Nardo-Marino et al., 2020). Evidence-based guidelines for SCD recommend use of shared decision-making (SDM), a collaborative process in which patients and clinicians work together to reach decisions by balancing scientific evidence with patient values, preferences, and treatment goals (Stiggelbout et al., 2012). SDM enhances patient knowledge, engagement, satisfaction, and adherence (Stacey et al., 2014; Wyatt et al., 2015) and is especially impactful for marginalized groups facing heightened barriers to inclusion in decision-making (Durand et al., 2014). Because adolescents and young adults (AYAs) with SCD demonstrate suboptimal use of disease-modifying therapies and increased risk for morbidity and mortality relative to other ages (Binder et al., 2013; Hamideh & Alvarez, 2013; Quinn et al., 2010), we created the SCD Shared Decision-Making Toolkit for AYAs (SDMT-AYA). To develop this toolkit, we modified an existing hydroxyurea decision support intervention for parents (Crosby et al., 2019) by obtaining input from AYAs with SCD, caregivers, and healthcare providers to expand the tools beyond hydroxyurea and address specific decision needs for AYAs (Ding et al., 2024; Hildenbrand et al., 2022; Hildenbrand et al., 2023). We report on refinement of SDMT-AYA and its acceptability and usability.

Methods: Iterative cycles of usability testing were conducted with AYAs, caregivers, and clinicians to establish technical and functional reliability of SDMT-AYA and the implementation blueprint. Usability sessions, including think-aloud testing and semi-structured interviews, were audio-recorded and transcribed. Rapid assessment procedures (Beebe, 2001) were used to synthesize think-aloud field notes and interview data into a summary matrix of prototype modifications, which were made in real-time before subsequent testing cycles.

Results: Through these iterative, user-centered design processes, our team refined SDMT-AYA. The toolkit targets multi-level barriers to use of disease-modifying therapies by providing technology-enhanced tools for: 1) AYAs and caregivers (i.e., multimedia decision aids, including videos, animations, and virtual reality); 2) clinicians (i.e., interactive training in SDM with AYAs, in-clinic conversation tools); and 3) clinic implementation (e.g., process mapping, audit and feedback). SDMT-AYA addresses key social determinants of health barriers for individuals with SCD (i.e., patient health literacy, provider bias, healthcare quality; Khan et al., 2023).

Usability testing was completed with 9 AYAs with SCD (15-25 years, M=19.1 years, 67% male, 89% Black or African American), 7 caregivers of AYAs (86% mothers, 100% Black or African American), and 10 hematology clinicians (80% female, 80% physicians, M=13.1 years in practice). AYAs and caregivers reported high satisfaction with decision aids, finding them trustworthy and engaging. Clinicians found the patient decision aids, training in SDM, and implementation support components acceptable. Think-aloud procedures revealed challenges navigating the website hosting patient decision aids and opportunities to improve clarity, interactivity, and portability of toolkit components. Modifications were made to improve website navigation; refinements to clinician training and implementation supports are ongoing.

Conclusions: Preliminary findings suggest high usability and acceptability of SDMT-AYA. A pilot trial is underway to examine feasibility and effectiveness of SDMT-AYA. Research to support broader dissemination and sustainment of evidence-based tools to support SDM in hematology care is essential to improve care quality and outcomes for AYAs with SCD.

Acknowledgements: Research was supported by the National Institute of General Medical Sciences of the National Institutes of Health (Award Number 2P20GM109021). The content is solely the responsibility of the authors and does not represent the official views of the National Institutes of Health.