Sickle Cell Disease, Sickle Cell Anemia, Hemoglobin SS, Hemoglobin SC, Hemoglobin Beta Thalassemia
Conditions
Keywords
Hydroxyurea, Bone marrow transplant, Chronic blood transfusion, Treatment options for sickle cell disease, Decision making
Brief 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 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. 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 that enable patients to make decisions based on their individual values and preferences.
Detailed description
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. There are minimal data about patient-related barriers to and attitudes towards, the use of curative therapies in SCD. 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. 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. The overarching goal of this project is to 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. 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 that enable patients to make decisions based on their individual values and preferences. Investigators hypothesize 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. The aims of the study are to estimate 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.
Interventions
OTHERDecision Aid Tool
The tool is a web based decision aid that provides information about the risks and benefits associated with sickle cell disease therapies. Participants will be provided a unique access ID and password to access the information.
OTHERStandard Practice
Standard of care teaching and discussion regarding treatment options given by patient's healthcare provider.
Sponsors
Patient-Centered Outcomes Research Institute
Emory University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
HEALTH_SERVICES_RESEARCH
Masking
SINGLE (Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
8 Years to 80 Years
Healthy volunteers
Yes
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Acceptability of Decision Aid Education Assessed by the Acceptability Survey | Post Visit 1 (Up to 2 Weeks) | Subjects will take an acceptability of education questionnaire which is a 8-item survey to assess the comprehension of education received for the decision aid tool. Each item will be scored on a scale from 1-4 where 1=poor, 2=fair, 3=good, and 4=excellent. Scores will be rated individually 1-4 according to each item. There is no overall total score. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Mean Difference in Decisional Conflict Scale Scores | Baseline, Month 3 | Decisional Conflict scale responses are scored for the total score, uncertainty sub-score, informed sub-score, values clarity sub-score, support sub-score and effective decision sub-score. The total score ranges from 0 (no decisional conflict) to 100 (extremely high decisional conflict). The uncertainty sub-score ranges from 0 (feels extremely certain about best choice) to 100 (feels extremely uncertain about best choice). The informed sub-score ranges from 0 (feels extremely informed) to 100 (feels extremely uninformed). The values clarity sub-score ranges from 0 (feels extremely clear about personal values for benefits & risks) to 100 (feels extremely unclear about personal values). The support sub-score ranges from 0 (feels extremely supported in decision making) to 100 (feels extremely unsupported in decision making). The effective decision sub-score ranges from 0 (good decision) to 100 (bad decision). |
| Mean Values Survey Score | Post Visit 1 (Up to 2 Weeks) | The values survey consists of 14 multiple choice questions to measure what is important to a patient when making decisions. The patient decision aid will be tested in the twelve domains of the international patient decision aid standards collaboration criteria checklist. Respondents will be asked to identify perceived importance of individual items (such as procedure related complications, decreasing complication risks, experiencing less pain) and to rate this importance on a 10 point likert scale (0-10) where 1 indicates not important to me at all and 10 indicates extremely important to me. Scores are then converted it to an 11 point scale and averaged. |
| Mean Decisional Self-Efficacy Scale Score | Month 3, Month 6 | The Decision Self-Efficacy Scale measures self-confidence or belief in one's ability to make decisions, including participate in shared decision making. Items are scored on a scale of 0-4 where 0 is not at all confident and 4 represents very confident.Total scores range from 0 (not at all confident) to 100 (very confident). A score of 0 means 'extremely low self- efficacy' and a score of 100 means 'extremely high self-efficacy. |
| Mean Knowledge Survey Scores | Baseline, Month 3, Month 6 | Knowledge Survey is a 25 multiple choice questionnaire which assesses how much knowledge is being retained after information about risks is received. The knowledge survey is scored as percent correct answers at each time point. This is a set of questions to test knowledge and understanding about sickle cell disease and treatments. As such the answers are dichotomous i.e true or false. The total score of percent correct answers is scored in the range of 0-100%. |
| Mean Decisional Regret Scale Score | Visit 3 | Decision Regret Scale measures distress or remorse after a health care decision. The subject rates regret using a 5 point Likert scale in answering the following questions; 1. It was the right decision 2. I regret the decision 3. I would go for the same decision if I were to do it again 4. The decision caused me a lot of harm 5. It was a wise decision. Total scores range from 0 to 100. A score of 0 means no regret; a score of 100 means high regret. |
| Mean Change in Preparation for Decision Making Scale Score | Month 3, Month 6 | Preparation for Decision Making Scale assesses a patient's perception of how useful a decision aid or other decision support intervention is in preparing the respondent to communicate with their practitioner at a consultation focused on making a health decision. The preparation for decision-making scale is scored on a 0-100 scale. Higher scores indicate a higher perceived level of preparation for decision making. The total score on the decision making scale is a continuous outcome. |
Countries
United States
Participant flow
Recruitment details
Participants were recruited between January 2015 and May 2016.
Pre-assignment details
Of the 134 participants who signed consent, 120 began study participation and were included in the baseline analysis.
Participants by arm
| Arm | Count |
|---|---|
| Standard Practice Participants received education regarding treatment consideration from their healthcare provider/team as per standard practice (usual care). | 60 |
| Standard Practice + Decision Aid Participants received standard of care teaching and discussion in addition to web-based decision aid tool access. | 60 |
| Total | 120 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Lost to Follow-up | 42 | 32 |
Baseline characteristics
| Characteristic | Standard Practice | Standard Practice + Decision Aid | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 60 Participants | 60 Participants | 120 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 60 Participants | 60 Participants | 120 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 0 Participants | 0 Participants | 0 Participants |
| Region of Enrollment United States | 60 Participants | 60 Participants | 120 Participants |
| Sex: Female, Male Female | 46 Participants | 45 Participants | 91 Participants |
| Sex: Female, Male Male | 14 Participants | 15 Participants | 29 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 60 | 0 / 60 |
| other Total, other adverse events | 0 / 60 | 0 / 60 |
| serious Total, serious adverse events | 0 / 60 | 0 / 60 |
Outcome results
Primary
Acceptability of Decision Aid Education Assessed by the Acceptability Survey
Subjects will take an acceptability of education questionnaire which is a 8-item survey to assess the comprehension of education received for the decision aid tool. Each item will be scored on a scale from 1-4 where 1=poor, 2=fair, 3=good, and 4=excellent. Scores will be rated individually 1-4 according to each item. There is no overall total score.
Time frame: Post Visit 1 (Up to 2 Weeks)
Population: Analysis was completed in both the standard practice and standard practice + decision aid groups together. There were a total of 106 participants who completed the survey.
| Arm | Measure | Group | Value (MEDIAN) | Dispersion |
|---|---|---|---|---|
| Standard Practice and Standard Practice + Decision Aid Groups | Acceptability of Decision Aid Education Assessed by the Acceptability Survey | Health Impact of Sickle Cell Disease | 3 units on a scale | Standard Deviation 0.86 |
| Standard Practice and Standard Practice + Decision Aid Groups | Acceptability of Decision Aid Education Assessed by the Acceptability Survey | Risk Factors | 3 units on a scale | Standard Deviation 0.97 |
| Standard Practice and Standard Practice + Decision Aid Groups | Acceptability of Decision Aid Education Assessed by the Acceptability Survey | Research | 3 units on a scale | Standard Deviation 1.1 |
| Standard Practice and Standard Practice + Decision Aid Groups | Acceptability of Decision Aid Education Assessed by the Acceptability Survey | Treatment Options | 3 units on a scale | Standard Deviation 1.1 |
| Standard Practice and Standard Practice + Decision Aid Groups | Acceptability of Decision Aid Education Assessed by the Acceptability Survey | Hydroxyurea/BMT/CBT | 3 units on a scale | Standard Deviation 1 |
| Standard Practice and Standard Practice + Decision Aid Groups | Acceptability of Decision Aid Education Assessed by the Acceptability Survey | Evidence About Hydroxyurea/BMT/CBT | 3 units on a scale | Standard Deviation 1.1 |
| Standard Practice and Standard Practice + Decision Aid Groups | Acceptability of Decision Aid Education Assessed by the Acceptability Survey | Stories About Others | 3 units on a scale | Standard Deviation 0.64 |
| Standard Practice and Standard Practice + Decision Aid Groups | Acceptability of Decision Aid Education Assessed by the Acceptability Survey | Evidence Supporting Self -Care | 3 units on a scale | Standard Deviation 1.1 |
Secondary
Mean Change in Preparation for Decision Making Scale Score
Preparation for Decision Making Scale assesses a patient's perception of how useful a decision aid or other decision support intervention is in preparing the respondent to communicate with their practitioner at a consultation focused on making a health decision. The preparation for decision-making scale is scored on a 0-100 scale. Higher scores indicate a higher perceived level of preparation for decision making. The total score on the decision making scale is a continuous outcome.
Time frame: Month 3, Month 6
Population: The analysis includes all participants who completed the survey.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Change in Preparation for Decision Making Scale Score | .48 units on a scale | Standard Deviation 0.89 |
| Standard Practice + Decision Aid | Mean Change in Preparation for Decision Making Scale Score | .38 units on a scale | Standard Deviation 0.74 |
Secondary
Mean Decisional Regret Scale Score
Decision Regret Scale measures distress or remorse after a health care decision. The subject rates regret using a 5 point Likert scale in answering the following questions; 1. It was the right decision 2. I regret the decision 3. I would go for the same decision if I were to do it again 4. The decision caused me a lot of harm 5. It was a wise decision. Total scores range from 0 to 100. A score of 0 means no regret; a score of 100 means high regret.
Time frame: Visit 3
Population: Number of participants who completed the scale.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Decisional Regret Scale Score | 27.5 units on a scale | Standard Deviation 23.2 |
Secondary
Mean Decisional Self-Efficacy Scale Score
The Decision Self-Efficacy Scale measures self-confidence or belief in one's ability to make decisions, including participate in shared decision making. Items are scored on a scale of 0-4 where 0 is not at all confident and 4 represents very confident.Total scores range from 0 (not at all confident) to 100 (very confident). A score of 0 means 'extremely low self- efficacy' and a score of 100 means 'extremely high self-efficacy.
Time frame: Month 3, Month 6
Population: Analysis was completed for participants who completed the scale at month 3 and month 6 visits.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Decisional Self-Efficacy Scale Score | Month 3 | 59.29 units on a scale | Standard Deviation 22.4 |
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Decisional Self-Efficacy Scale Score | Month 6 | 71.36 units on a scale | Standard Deviation 29.97 |
| Standard Practice + Decision Aid | Mean Decisional Self-Efficacy Scale Score | Month 3 | 75.31 units on a scale | Standard Deviation 22.1 |
| Standard Practice + Decision Aid | Mean Decisional Self-Efficacy Scale Score | Month 6 | 84.84 units on a scale | Standard Deviation 16.39 |
Secondary
Mean Difference in Decisional Conflict Scale Scores
Decisional Conflict scale responses are scored for the total score, uncertainty sub-score, informed sub-score, values clarity sub-score, support sub-score and effective decision sub-score. The total score ranges from 0 (no decisional conflict) to 100 (extremely high decisional conflict). The uncertainty sub-score ranges from 0 (feels extremely certain about best choice) to 100 (feels extremely uncertain about best choice). The informed sub-score ranges from 0 (feels extremely informed) to 100 (feels extremely uninformed). The values clarity sub-score ranges from 0 (feels extremely clear about personal values for benefits & risks) to 100 (feels extremely unclear about personal values). The support sub-score ranges from 0 (feels extremely supported in decision making) to 100 (feels extremely unsupported in decision making). The effective decision sub-score ranges from 0 (good decision) to 100 (bad decision).
Time frame: Baseline, Month 6
Population: Analysis was completed for participants who completed the scale at both baseline and month 6 visits.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Difference in Decisional Conflict Scale Scores | Total Score | 4.6 units on a scale |
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Difference in Decisional Conflict Scale Scores | Uncertainty | 7.2 units on a scale |
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Difference in Decisional Conflict Scale Scores | Informed | 1.7 units on a scale |
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Difference in Decisional Conflict Scale Scores | Values Clarity | 6.7 units on a scale |
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Difference in Decisional Conflict Scale Scores | Support | .6 units on a scale |
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Difference in Decisional Conflict Scale Scores | Effective Decision | 6.4 units on a scale |
| Standard Practice + Decision Aid | Mean Difference in Decisional Conflict Scale Scores | Support | 1.3 units on a scale |
| Standard Practice + Decision Aid | Mean Difference in Decisional Conflict Scale Scores | Total Score | -5.0 units on a scale |
| Standard Practice + Decision Aid | Mean Difference in Decisional Conflict Scale Scores | Values Clarity | -11.7 units on a scale |
| Standard Practice + Decision Aid | Mean Difference in Decisional Conflict Scale Scores | Uncertainty | .8 units on a scale |
| Standard Practice + Decision Aid | Mean Difference in Decisional Conflict Scale Scores | Effective Decision | -2.4 units on a scale |
| Standard Practice + Decision Aid | Mean Difference in Decisional Conflict Scale Scores | Informed | 10.0 units on a scale |
Secondary
Mean Difference in Decisional Conflict Scale Scores
Decisional Conflict scale responses are scored for the total score, uncertainty sub-score, informed sub-score, values clarity sub-score, support sub-score and effective decision sub-score. The total score ranges from 0 (no decisional conflict) to 100 (extremely high decisional conflict). The uncertainty sub-score ranges from 0 (feels extremely certain about best choice) to 100 (feels extremely uncertain about best choice). The informed sub-score ranges from 0 (feels extremely informed) to 100 (feels extremely uninformed). The values clarity sub-score ranges from 0 (feels extremely clear about personal values for benefits & risks) to 100 (feels extremely unclear about personal values). The support sub-score ranges from 0 (feels extremely supported in decision making) to 100 (feels extremely unsupported in decision making). The effective decision sub-score ranges from 0 (good decision) to 100 (bad decision).
Time frame: Baseline, Month 3
Population: Analysis was completed for participants who completed the scale at both baseline and month 3 visits.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Difference in Decisional Conflict Scale Scores | Support | -.4 units on a scale |
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Difference in Decisional Conflict Scale Scores | Values Clarity | -.001 units on a scale |
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Difference in Decisional Conflict Scale Scores | Uncertainty | .7 units on a scale |
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Difference in Decisional Conflict Scale Scores | Informed | -1.3 units on a scale |
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Difference in Decisional Conflict Scale Scores | Effective Decision | 1.1 units on a scale |
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Difference in Decisional Conflict Scale Scores | Total Score | 1.0 units on a scale |
| Standard Practice + Decision Aid | Mean Difference in Decisional Conflict Scale Scores | Effective Decision | 2.3 units on a scale |
| Standard Practice + Decision Aid | Mean Difference in Decisional Conflict Scale Scores | Total Score | 4.8 units on a scale |
| Standard Practice + Decision Aid | Mean Difference in Decisional Conflict Scale Scores | Uncertainty | 1.9 units on a scale |
| Standard Practice + Decision Aid | Mean Difference in Decisional Conflict Scale Scores | Informed | -12.1 units on a scale |
| Standard Practice + Decision Aid | Mean Difference in Decisional Conflict Scale Scores | Values Clarity | -8.0 units on a scale |
| Standard Practice + Decision Aid | Mean Difference in Decisional Conflict Scale Scores | Support | 4.6 units on a scale |
Secondary
Mean Knowledge Survey Scores
Knowledge Survey is a 25 multiple choice questionnaire which assesses how much knowledge is being retained after information about risks is received. The knowledge survey is scored as percent correct answers at each time point. This is a set of questions to test knowledge and understanding about sickle cell disease and treatments. As such the answers are dichotomous i.e true or false. The total score of percent correct answers is scored in the range of 0-100%.
Time frame: Baseline, Month 3, Month 6
Population: The analysis includes all participants who completed the survey.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Knowledge Survey Scores | Baseline | 38 units on a scale | Standard Deviation 49.87 |
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Knowledge Survey Scores | Month 3 | 19 units on a scale | Standard Deviation 52.9 |
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Knowledge Survey Scores | Month 6 | 46.79 units on a scale | Standard Deviation 51.18 |
| Standard Practice + Decision Aid | Mean Knowledge Survey Scores | Baseline | 39 units on a scale | Standard Deviation 49.83 |
| Standard Practice + Decision Aid | Mean Knowledge Survey Scores | Month 3 | 23 units on a scale | Standard Deviation 52.9 |
| Standard Practice + Decision Aid | Mean Knowledge Survey Scores | Month 6 | 22 units on a scale | Standard Deviation 55.54 |
Secondary
Mean Values Survey Score
The values survey consists of 14 multiple choice questions to measure what is important to a patient when making decisions. The patient decision aid will be tested in the twelve domains of the international patient decision aid standards collaboration criteria checklist. Respondents will be asked to identify perceived importance of individual items (such as procedure related complications, decreasing complication risks, experiencing less pain) and to rate this importance on a 10 point likert scale (0-10) where 1 indicates not important to me at all and 10 indicates extremely important to me. Scores are then converted it to an 11 point scale and averaged.
Time frame: Post Visit 1 (Up to 2 Weeks)
Population: The analysis includes all participants who completed the survey.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Standard Practice and Standard Practice + Decision Aid Groups | Mean Values Survey Score | 10.2 units on a scale | Standard Deviation 2 |
| Standard Practice + Decision Aid | Mean Values Survey Score | 10 units on a scale | Standard Deviation 1.7 |