BACKGROUND

Hydroxyurea, chronic blood transfusion, and bone marrow transplantation can modify sickle cell disease (SCD), reduce complications, and improve survival; however, these treatments are associated with a significant decisional dilemma because of the inherent risk–benefit tradeoffs and the lack of comparative studies. These treatments are underutilized, leading to avoidable morbidity and premature mortality. A web-based decision aid has the potential to provide patients high-quality information about their treatment options and the associated risks and benefits, help them clarify their values, and allow them to share in the process of informed medical decision-making.

OBJECTIVES

Aim 1 was to develop a health literacy–sensitive, web-based decision aid to help patients with SCD make informed decisions about treatment. Aim 2 was to estimate in a randomized clinical trial (RCT) the effectiveness of this decision aid, which was tailored to patient characteristics in improving patient knowledge, patient involvement in decision-making, and decision-making quality.

METHODS

Following the Ottawa Decision Support Framework, we conducted an SCD population decisional needs assessment to guide the development of a web-based decision aid. A nationwide sample of patients, caregivers, stakeholders, and health care providers (n = 223) participated in qualitative interviews to identify decisional conflict (uncertainty); knowledge and expectations; values (what is important to patients); support and resources; decision types, timing, stages, and learning; and personal clinical characteristics. Transcripts were coded using QSR NVivo 10 (https://www.qsrinternational.com/nvivo/home). We developed a web-based decision aid (http://sickleoptions.org/), and alpha testing by 30 patients and 38 health care providers established its accuracy and usability. Another group of stakeholders (n = 87) completed iterative cycles of beta testing. We conducted an RCT of adults and of caregivers of pediatric patients to evaluate the decision aid's efficacy.

RESULTS

In an RCT of 120 participants (60 patients and 60 controls), only 39 in each group completed the baseline survey and only 43 of the total 120 completed a 3-month survey; during the course of the study, only 7 individuals made a treatment decision. Given this paucity of trial data, results from the RCT could not be considered conclusive; however, qualitative interviews revealed high levels of usability, acceptability, and utility in education, values clarification, and preparation for decision-making. Further, the decision aid met international patient decision aid collaboration standards for content, development process, and efficacy—with the exception of having a full range of positive and negative experiences in patient stories.

CONCLUSIONS

In the RCT, survey data were insufficient to draw any conclusions about the impact of the decision aid; however, the qualitative data suggest that patients with SCD found the decision aid to be acceptable, useful, and feasible.

LIMITATIONS

The large amount of missing survey data limits drawing conclusions about the effectiveness of the decision aid in improving patient involvement in decision-making and in decision-making quality.