Comparative Effectiveness of a Web-Based Patient Decision Aid for Therapeutic Options for Sickle Cell Disease: Randomized Controlled Trial

Abstract

BackgroundHydroxyurea, chronic blood transfusions, and bone marrow transplantation are efficacious, disease-modifying therapies for sickle cell disease but involve complex risk-benefit trade-offs and decisional dilemma compounded by the lack of comparative studies. A patient decision aid can inform patients about their treatment options, the associated risks and benefits, help them clarify their values, and allow them to participate in medical decision making.ObjectiveThe objective of this study was to develop a literacy-sensitive Web-based patient decision aid based on the Ottawa decision support framework, and through a randomized clinical trial estimate the effectiveness of the patient decision aid in improving patient knowledge and their involvement in decision making.MethodsWe conducted population decisional needs assessments in a nationwide sample of patients, caregivers, community advocates, policy makers, and health care providers using qualitative interviews to identify decisional conflict, knowledge and expectations, values, support and resources, decision types, timing, stages and learning, and personal clinical characteristics. Interview transcripts were coded using QSR NVivo 10. Alpha testing of the patient decision aid prototype was done to establish usability and the accuracy of the information it conveyed, and then was followed by 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 patient decision aid.ResultsIn a decisional needs assessment, 223 stakeholders described their preferences, helping to guide the development of the patient decision aid, which then underwent alpha testing by 30 patients and 38 health care providers and iterative cycles of beta testing by 87 stakeholders. In a randomized clinical trial, 120 participants were assigned to either the patient decision aid or standard care (SC) arm. Qualitative interviews revealed high levels of usability, acceptability, and utility of the patient decision aid in education, values clarification, and preparation for decision making. On the acceptability survey, 72% (86/120) of participants rated the patient decision aid as good or excellent. Participants on the patient decision aid arm compared to the SC arm demonstrated a statistically significant improvement in decisional self-efficacy (P=.05) and a reduction in the informed sub-score of decisional conflict (P=.003) at 3 months, with an improvement in preparation for decision making (P<.001) at 6 months. However, there was no improvement in terms of the change in knowledge, the total or other domain scores of decisional conflicts, or decisional self-efficacies at 6 months. The large amount of missing data from survey completion limited our ability to draw conclusions about the effectiveness of the patient decision aid. The patient decision aid met 61 of 62 benchmarks of the international patient decision aid collaboration standards for content, development process, and efficacy.Conclusions...

Assessment of Patient and Caregiver Attitudes and Approaches to Decision-Making Regarding Bone Marrow Transplant for Sickle Cell Disease

“…Study participants included individuals aged 18 years or older with SCD and caregivers of patients with SCD enrolled in the parent study to develop and test a web-based decision aid for SCD. 19 Participants were drawn from a convenience sample recruited from national and regional conferences, and local clinics in 2 cities. For this analysis, we used interview transcripts from the needs assessment phase in 2013 to 2014 (group 1) and from the baseline of the randomized clinical trial phase in 2015 to 2016 (group 2).…”

“…Details related to interview methods and script have been previously published. 19 We identified all transcripts that contained the terms bone marrow, BMT, stem cell, transplant, bone marrow transplant, or cure. All transcripts that met criteria were included.…”

“…Interviews used a semistructured format with probes for follow-up, but the exact wording, order, or content of questions may have differed between participants, and some concepts were explored more with some participants than others. The parent study 19 was approved by the Emory University institutional review board and University of Pittsburgh institutional review board, and participants provided informed consent. This study followed the Standards for Reporting Qualitative Research (SRQR) 20 reporting guideline for qualitative studies.…”

“…18 This study is a theoryinformed secondary analysis of a subset of interview transcripts from a parent study, a study to develop a web-based patient decision aid for therapeutic options in SCD and evaluate its effectiveness in a randomized clinical trial. 19…”

Noninvasive Hemoglobin Level Prediction in a Mobile Phone Environment: State of the Art Review and Recommendations

“…Approximately 5.6% of the US population is anemic and 1.5% of the population has moderate to severe anemia [5]. Sickle cell diseases (SCD) cost more than US $1.5 billion annually in the United States [6]. Globally, blood disorders and associated complications affect more than 5 million people.…”