Implementation of Shared Decision-Making for Sickle Cell Disease (Implement-SDM Study) - Modified Project Summary/Abstract Section Sickle cell disease (SCD) is a group of genetic hemoglobin disorders that impacts approximately 100,000 individuals in the United States, leading to significant health challenges and early mortality. New and transformative disease-modifying treatments for pediatric SCD have allowed for more personalized disease management. However, choosing the right treatment has become more difficult due to the varied mechanisms of action, eligibility criteria, safety and efficacy profiles, and administration methods of these treatments (hydroxyurea is a daily oral medication with unknown long-term side effects, crizanlizumab is administered through monthly infusions, L-glutamine is a daily liquid dispersion, and gene therapy requires chemotherapy+infusion+1yr monitoring). Additionally, subjective clinical judgement and patient mistrust impact treatment decisions, making it vital for patients and families to be actively involved in these discussions. Unfortunately, clinicians often lack the training and resources to effectively incorporate patient preferences into treatment decisions, resulting in low uptake and leaving many patients to endure SCD complications. Shared decision-making (SDM), a clinical practice that integrates medical evidence with patient preferences, is recommended when multiple factors influence treatment selection and for chronic conditions with intensive treatments. Yet, SDM remains understudied and underutilized in pediatric SCD. To address this gap, our team developed a Shared Decision-Making Toolkit (SDMT) focusing on patient/caregiver education and provider engagement, initially targeting hydroxyurea. Our pilot study comparing the SDMT to usual care found increased reach (from 33% to 100% offered hydroxyurea) and lower decisional conflict among parents (d=0.41). Next, we conducted a study to disseminate the toolkit to nine pediatric SCD clinics. Despite most sites failing to achieve full SDMT implementation, the adoption rate was 59%. In this project, we will refine the SDMT to address all treatment options and all age groups (0-21 years) (Aim 1). We will then conduct a type II hybrid effectiveness-implementation trial to evaluate the refined SDMT's impact on patient/caregiver perception of SDM (primary outcome) and clinical outcomes (Aim 2). We will also assess the feasibility of a rigorous implementation strategy, the Learning Community, on adoption of SDM by measuring documentation of SDM in medical records (secondary outcome; Aim 3). Utilizing mixed methods, we will identify contextual factors and predictors influencing successful SDMT implementation and develop an implementation blueprint/guide for dissemination. This work has the potential to transform SCD care and improve health outcomes. Additionally, participation in the Learning Community will meet Maintenance Of Certification (MOC) Part 4 activity guidelines, and the implementation guide will contain billing support for SDM. Thus, the project and implementation guide could be adapted for use with other chronic conditions.
