Overview

This trial is active, not recruiting.

Conditions sickle cell disease, sickle cell anemia, hemoglobin ss, hemoglobin sc, hemoglobin beta thalassemia
Treatments decision aid, standard practice
Sponsor Emory University
Collaborator Patient-Centered Outcomes Research Institute
Start date September 2014
End date December 2016
Trial size 120 participants
Trial identifier NCT02326597, IRB00076096

Summary

Sickle cell disease (SCD) is an inherited disorder with chronic multi-system manifestations affecting 100,000 individuals in the US, largely of minority origin and associated with substantial morbidity, premature mortality, individual suffering, healthcare costs and loss of productivity. Disease modifying treatments such as Hydroxyurea, chronic blood transfusion and curative bone marrow transplantation are offered to patients based on physician preference and current practice informed by clinical trials. Decision aids are tools that could help translate evidence from these sources into practice by helping clinicians involve patients in making deliberate choices based on accessible information about the options available and their outcomes and to help them make decisions based on their values and preferences.

The overarching goal of this project is to develop and implement a web based decision aid individualized to patient characteristics to help patients with SCD achieve more accurate perception of risks and benefits of treatment options and make decisions in congruence with their values and preferences.

The investigators will conduct needs assessment interviews as indicated by the data for saturation and validation of findings. The investigators will conduct beta testing of the web-based decision aid to clarify development and validate findings. The investigators will use a randomized controlled trial of the effectiveness of a web-based decision aid to give patients accurate information about risks and benefits of therapies and enable them to make decisions based on their individual values and preferences.

To provide accurate information to patients and caregivers about the risks and benefits of various treatments for sickle cell disease and to enable them to make informed and involved therapeutic decisions based on their values, preferences and stated goals.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification efficacy study
Intervention model parallel assignment
Masking single blind (outcomes assessor)
Primary purpose supportive care
Arm
(Active Comparator)
Standard care teaching and discussion. This group will crossover to be offered the opportunity to visit the Decision Aid website after the final study visits/surveys and data collection of the intervention group (standard practice plus decision aid).
decision aid
Use of decision aid tool to aid in understanding treatment option and identifying personal values and beliefs accessible via devices: iPad, Smart phone, Computer, Tablet
standard practice
Standard care teaching and discussion in addition to web-based decision aid tool accessible via devices: iPad, Smart phone, Computer, Tablet
(Experimental)
Standard care teaching and discussion in addition to web-based decision aid tool access
decision aid
Use of decision aid tool to aid in understanding treatment option and identifying personal values and beliefs accessible via devices: iPad, Smart phone, Computer, Tablet
standard practice
Standard care teaching and discussion in addition to web-based decision aid tool accessible via devices: iPad, Smart phone, Computer, Tablet

Primary Outcomes

Measure
Acceptability of education change from Baseline Visit to Visit 2 to Visit 3 to Visit 4
time frame: Baseline (V1), 3-4 mths (V2) and 6-8 mths (V3) 8-11 mths (V4) for crossover group

Secondary Outcomes

Measure
Decision Self-Efficacy Scale change from Baseline Visit to Visit 2 to Visit 3 to Visit 4
time frame: Baseline (V1), 3-4 mths (V2) and 6-8 mths (V3) 8-11 mths (V4) for crossover group
Decisional Conflict Scale change from Baseline Visit to Visit 2 to Visit 3 to Visit 4
time frame: Baseline (V1), 3-4 mths (V2) and 6-8 mths (V3) 8-11 mths (V4) for crossover group
Values survey change from Baseline Visit to Visit 2 to Visit 3 to Visit 4
time frame: Baseline (V1), 3-4 mths (V2) and 6-8 mths (V3) 8-11 mths (V4) for crossover group
Realistic Expectations change from Baseline Visit to Visit 2 to Visit 3 to Visit 4
time frame: Baseline (V1), 3-4 mths (V2) and 6-8 mths (V3) 8-11 mths (V4) for crossover group
Preparation for Decision Making Scale change from Baseline Visit to Visit 2 to Visit 3 to Visit 4
time frame: Baseline (V1), 3-4 mths (V2) and 6-8 mths (V3) 8-11 mths (V4) for crossover group
Knowledge Survey change from Baseline Visit to Visit 2 to Visit 3 to Visit 4
time frame: Baseline (V1), 3-4 mths (V2) and 6-8 mths (V3) 8-11 mths (V4) for crossover group
Choice Predisposition and Decision change from Baseline Visit to Visit 2 to Visit 3 to Visit 4
time frame: Baseline (V1), 3-4 mths (V2) and 6-8 mths (V3) 8-11 mths (V4) for crossover group
Stage of Decision Making change from Baseline Visit to Visit 2 to Visit 3 to Visit 4
time frame: Baseline (V1), 3-4 mths (V2) and 6-8 mths (V3) 8-11 mths (V4) for crossover group
Decision Regret Scale change from Baseline Visit to Visit 2 to Visit 3 to Visit 4
time frame: Baseline (V1), 3-4 mths (V2) and 6-8 mths (V3) 8-11 mths (V4) for crossover group

Eligibility Criteria

Male or female participants from 8 years up to 80 years old.

Inclusion Criteria: - Individuals with sickle cell disease ages 8 to 80 years, inclusive OR - Parent/legal guardian of patients (age < 18 years) with sickle cell disease who are directly involved in decision making regarding sickle cell disease healthcare treatment OR - Health care provider directly involved in care of individuals with sickle cell disease, including child of parent/legal guardian enrolled in study - Patients/parents/caregivers who have made a past decision to not obtain treatment of the considered option or who have not obtained treatment of the chosen option in past 12 months. - All participants will be able to comprehend English - Patients/Parent/Legal guardian will have access to the internet from iPad, smart phone or personal computer Exclusion Criteria: - Family Members/Individuals/Caregivers not directly involved in decision-making regarding sickle cell disease healthcare. - Patient/parent/legal guardian who has already made a decision to begin and has started the treatment option. - Child < 18 years of parent/legal guardian who is participating in Cohort A of this study and randomized to the control arm and not the decision aid arm. - Spouse, significant other, or other family member involved in decision making for child <18 years if parent/legal guardian of child already enrolled into this study.

Additional Information

Official title Comparative Effectiveness of a Decision Aid for Therapeutic Options in Sickle Cell Disease
Principal investigator Lakshmanan Krishnamurti, MD
Description Sickle cell disease (SCD) is a major public health problem in the United States with an estimated 100,000 individuals living with this disease. It is associated with significant morbidity caused by pain crises, acute chest syndrome, stroke, pulmonary hypertension, leg ulcers and irreversible organ damage. Quality of life is impaired in children and adults with this disease 3-6 and productivity is impaired, with a 40-60% unemployment rate in adult patients. The disease results in premature mortality, with a median age of death of 38 years. In 2010, there were approximately 100,000 hospital discharges in the United States for which SCD was the principal diagnosis. Mean hospital charges per admission were $15,000, with the total annual charges in the US exceeding $1.5 billion. Approximately 70% of the patients were on government sponsored medical insurance, predominantly Medicaid and Medicare. Sickle cell disease is thus the archetype of the chronic multisystem disease predominantly affecting a minority, disadvantaged population. Hydroxyurea (HU) has been demonstrated in placebo controlled multi-center clinical trials to be efficacious in reducing complications such as vasocclusive pain crises and acute chest syndrome in children and adults with SCD and in improving survival in adults. It is the only disease-modifying therapy and the only drug approved by the FDA for use in patients with SCD. Hematopoietic stem cell transplantation has been demonstrated to be curative in patients with SCD. Only a minority of patients who meet eligibility criteria for transplantation, however, proceed with the treatment even when a matched sibling donor is available. The low utilization of curative strategies for SCD appears to be partly attributable to a lack of information about the technologies available to facilitate transplantation. There are minimal data about patient-related barriers to and attitudes towards, the use of curative therapies in SCD. A systematic review of the research on barriers to care in patients with SCD did not identify any studies that specifically addressed patient-related barriers to the use of Hydroxyurea. The attitudes of patients with SCD toward the use of hydroxyurea (HU) therapy may contribute to the underutilization of HU in the United States, yet our understanding of these attitudes is limited. Of patients who have never taken HU, half reported receiving no information about HU, and 85% report believing that they would receive no improvement if they were to take HU. In making choices regarding transfusion, transplantation or HU, families are most influenced by perceived efficacy and safety. Thus significant gaps remain in the understanding of patient perspectives, in the provision of accurate information about risks and benefits of therapies and of incorporating patients' values and preferences in offering treatment options. Thus, there is a need for research that helps to understand patient values and preferences and determines how to help patients make informed treatment decision in congruence with their values and preferences. Hypothesis The investigators overarching hypothesis is that a web based decision aid individualized to patient characteristics can improve knowledge and help patients with SCD achieve more accurate perception of risks and benefits of treatment options and is associated with lower decisional conflict than standard care. To test the hypothesis the investigators propose the following specific aims: Aim 1: Develop a health literacy sensitive, web based, decision aid to help patients with sickle cell disease make informed choices about treatment options such as hydroxyurea, chronic blood transfusion, bone marrow transplantation and standard comprehensive care. Aim 2: To estimate in a randomized clinical trial the effectiveness of the decision aid tailored to individual patient characteristics on patient knowledge, patient involvement in decision-making and decision-making quality, when compared with usual care. Research Questions: 1. What are the needs of patients with sickle cell disease and their parents/caregivers when considering treatment options for sickle cell disease? 2. Does the use of a decision aid result in improvement of patient knowledge, patient involvement in the decision, the congruence of their decision with their values and preferences? Methods Study Design for Qualitative Interviews Decisional Needs Assessment: Consent in-person or via telephone and 30-45 minute interview using semi-structured open-ended question guide developed based on Ottawa Decision Support Framework workbook, Decisional Needs Assessment in Populations. Interviews audio recorded, transcribed verbatim and analyzed for merging themes. Demographics obtained include but not limited to age, gender, education level, role in sickle cell disease. Data results will inform development of a web-based decision aid tool. Study Design for Beta Testing: Consent in-person or via telephone and 30-45 minute interview using semi-structured open-ended question guide developed based on cognitive interview techniques while web-based decision aid tool is viewed via internet using a computer or tablet. Demographics obtained include but not limited to age, gender, education level, role in sickle cell disease. Data results will inform editing and revision of the web-based decision aid tool. Study Design for Testing Decision Aid: Cohort A: a randomized controlled trial using mixed methods for the use of a web based decision aid to give patients with sickle cell disease and parent/legal guardian of children with sickle cell disease accurate information about risks and benefits of therapies and enable them to make decisions based on their individual values and preferences. Participants will complete surveys before, soon after and within 4 months following discussion with an health care provider to determine impact of the decision aid on knowledge, involvement in the decision and on decisional conflict, quality and uptake and adherence with the therapies. Qualitative semi-structured interviews will be collected to gather additional information related to decision making. The control group will participate in a cross-over design, receiving the decision aid tool after completion of the initial phase of the study. An additional qualitative interview will be collected regarding decision making. Cohort B: a mixed methods descriptive study design to determine the impact of the decision aid on quality and comprehension of information, and impact on daily life relating to management of sickle cell disease in patients <18 years of age. Cohort C - To better understand the effect of the decision aid in decision making and patient involvement as reported by the physicians consenting these patients (child and adult) for treatment will be asked to participate in the study. Health care providers will not be randomized.
Trial information was received from ClinicalTrials.gov and was last updated in August 2016.
Information provided to ClinicalTrials.gov by Emory University.