Partnering with adolescents and young adults with sickle cell disease in treatment decisions - sickle cell disease (SCD) is a life-threatening condition marked by debilitating complications, poor…

The National Institute of Nursing Research, part of the Department of Health and Human Services National Institutes of Health, awarded $789,558 to The Nemours Foundation on September 1, 2026, under the Nursing Research program (CFDA 93.361).

The award funds development and evaluation of the Sickle Cell Disease Shared Decision-Making Toolkit for Adolescents and Young Adults (SDMT-AYA), which addresses barriers to uptake and adherence to disease-modifying therapies among adolescents and young adults with sickle cell disease. The toolkit employs shared decision-making approaches to increase patient knowledge, engagement, and treatment adherence, targeting clinical, patient, and healthcare access barriers that currently result in suboptimal therapy adoption—approximately 25 percent of the population receives hydroxyurea treatment, and uptake of other therapies is estimated below 5 percent. The intervention provides tools for multidisciplinary clinical teams to support informed treatment decision-making.

The Nemours Foundation's Nemours Biomedical Research—Delaware Division, doing business as Nemours Children's Hospital, Delaware, is the recipient. Work is performed at Wilmington, Delaware. The period of performance runs from September 1, 2026, through June 30, 2031. The award is classified as a Project Grant, the standard assistance type under the Nursing Research program.

Partnering with adolescents and young adults with sickle cell disease in treatment decisions - sickle cell disease (SCD) is a life-threatening condition marked by debilitating complications, poor quality of life, and early mortality. Historically, SCD has received far less funding and public policy efforts than other chronic diseases, resulting in decades of stagnation in medical advances. However, treatment options for SCD are now expanding with recent federal and industry investments. For the first time, patients living with SCD are making decisions about multiple disease-modifying therapies, each with unique clinical indications and tradeoffs impacting quality of life. Unfortunately, most do not reap the benefits of these advances. Hydroxyurea is the mainstay treatment, yet only about 25% receive IT; uptake of other therapies is estimated at less than 5% of the population. Use of therapies is especially suboptimal among adolescents and young adults (AYAS) due to patient (lack of involvement in decisions, inadequate knowledge), provider (not offering HU due to concerns about AYAS' adherence), and healthcare access challenges (limited transportation, inadequate insurance). In turn, suboptimal use of therapies contributes to high morbidity and mortality among AYAS with SCD. Health promotion interventions that can effectively overcome these barriers are urgently needed to optimize uptake and adherence to SCD therapies among AYAS and thereby reduce preventable disease complications. Shared decision-making (SDM) enhances patient knowledge, engagement and satisfaction with care, adherence to treatments, and health outcomes. However, SDM is difficult to achieve in routine care, and there are no interventions that support SDM for AYAS with SCD. To address this unmet need, we developed the SCD shared decision-making toolkit for AYAS (SDMT-AYA). SDMT-AYA targets multi-level barriers to use of disease-modifying therapies by providing tools for: 1) multidisciplinary clinicians (skills-based training, communication support tools used at point-of-care); 2) AYAS and caregivers (decision aids that leverage advanced learning technologies); and 3) implementation (process mapping, audit and feedback). Preliminary findings suggest high usability, acceptability, and feasibility of the toolkit. This stepped-wedge cluster randomized trial will evaluate effectiveness of SDMT-AYA on patient experiences of SDM, patient knowledge of SCD and treatment options, and clinical outcomes (use of therapies, acute care utilization), test mechanisms of action, and examine barriers and facilitators to implementing and sustaining SDMT-AYA. Four sites will be randomized to cross over from usual care (use of a pocket guide for disease-modifying therapy discussions) to the intervention condition (use of SDMT-AYA to discuss disease-modifying therapies) at distinct time points. We will interview key informants at each site to explore barriers and facilitators affecting implementation and sustainment of the toolkit. This research has the potential to reinvent how healthcare systems educate, engage, and partner with AYAS in making care decisions. If successful, this project can serve as a model for facilitating SDM to improve care and outcomes for AYAS with other chronic conditions.