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.ConclusionsWe have developed a patient decision aid for sickle cell disease with extensive input from stakeholders and in a randomized clinical trial demonstrated its acceptability and utility in education and decision making. We were unable to demonstrate its effectiveness in improving patient knowledge and involvement in decision making.Trial RegistrationClinicalTrials.gov NCT03224429; https://clinicaltrials.gov/ct2/show/NCT03224429 and ClinicalTrials.gov NCT02326597; https://clinicaltrials.gov/ct2/show/NCT02326597
Highlights
Sickle cell disease (SCD) is a major public health problem in the United States, affecting an estimated 100,000 individuals [1] and associated with significant morbidity caused by pain crises, acute chest syndrome, stroke, pulmonary hypertension, leg ulcers, and irreversible organ damage [2]
We included: (1) individuals with SCD aged 8-80 years old; (2) individuals who were post bone marrow transplant for SCD; (3) parents/legal guardians/caregivers of individuals with SCD directly involved in decision making with/for that individual; (4) parents/legal guardians/caregivers of individuals who were post-bone marrow transplant for SCD who were directly involved in decision making with/for that individual; (5) stakeholders involved in any aspect of SCD; (6) health care providers who were directly involved in sickle cell health care, including but not limited to physicians, nurse practitioners, https://www.jmir.org/2019/12/e14462
J Med Internet Res 2019 | vol 21 | iss. 12 | e14462 | p. 5 decisional needs assessment about decision making for treatments for sickle cell disease (Table 1)
Summary
Sickle cell disease (SCD) is a major public health problem in the United States, affecting an estimated 100,000 individuals [1] and associated with significant morbidity caused by pain crises, acute chest syndrome, stroke, pulmonary hypertension, leg ulcers, and irreversible organ damage [2]. Disease-modifying therapies, such as hydroxyurea (HU), chronic transfusion therapy (CTT), and bone marrow transplantation (BMT), have demonstrated efficacy in clinical trials. There are trade-offs between the benefits and harms between the different treatment options, such that an individual patient’s preference, values, and risks of different outcomes could influence their decisions. A major contributor to decisional dilemma associated with treatments for SCD is the absence of studies to compare the benefits and harms of these treatments and guide patients in their choice of treatments. Hydroxyurea, 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
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