JMIR Publications

ABSTRACT

Background:

Hydroxyurea, chronic blood transfusion, and bone marrow transplantation are efficacious disease-modifying therapies for sickle cell disease (SCD) but are associated with a significant decisional dilemma because of the inherent risk-benefit tradeoffs and the lack of comparative studies. A web-based patient decision aid (PtDA) has the potential to provide patients with high-quality information about their treatment options and associated risks and benefits, help them clarify their values, and allow them to share in the process of informed medical decision making.

Objective:

The objective of this study was to develop a literacy-sensitive, web-based, PtDA using the conceptual framework of the Ottawa decision support framework, and to estimate in a randomized clinical trial the effectiveness of the PtDA in improving patient knowledge, and involvement in decision-making.

Methods:

We conducted population decisional needs assessment in a nationwide sample of patients, caregivers, stakeholders, and health care providers using 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. Interview transcripts were coded using QSR NVivo 10. Prototype PtDA underwent Alpha testing to establish usability and the accuracy of the information that it conveyed. Stakeholders participated in iterative cycles of beta testing. We conducted a randomized clinical trial of adults and of caregivers of pediatric patients to evaluate the efficacy of the PtDA.

Results:

A total of 223 stakeholders participated in decisional needs assessment and provided their preferences which guided the development of the PtDA (www.sickleoptions.org) which was then refined and finalized with alpha testing by 30 patients and 38 healthcare providers, and iterative cycles of beta testing by 87 stakeholders. To evaluate the efficacy of the PtDA, we enrolled 120 participants (60 in the decision aid and 60 in the usual care arm) in a randomized clinical trial. Qualitative interviews revealed high levels of usability, acceptability, and utility in education, values clarification, and preparation for decision making. The PtDA met most of the international patient decision aid collaboration standards for content, development process, and efficacy. While statistically significant improvement was observed in decisional self-efficacy and preparation for decision-making, as well reduction in overall decisional conflict score, informed sub-score and values clarification sub-score, the large amount of missing data in the completion of follow-up surveys limits the ability to draw conclusions about the effectiveness of the decision aid in improving patient knowledge and involvement in decision-making.

Conclusions:

We have developed a PtDA for SCD with extensive input from stakeholders. Qualitative data and surveys established the acceptability and utility of the PtDA in education and, decision making, but missing survey data limit conclusions about the effectiveness of the PtDA in Improving patient knowledge and involvement in decision making. Clinical Trial: NCT02326597