Trial Outcomes & Findings for Decision Aid for Therapeutic Options In Sickle Cell Disease (NCT NCT02326597)

NCT ID: NCT02326597

Last Updated: 2018-10-09

Results Overview

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.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

134 participants

Primary outcome timeframe

Post Visit 1 (Up to 2 Weeks)

Results posted on

2018-10-09

Participant Flow

Participants were recruited between January 2015 and May 2016.

Of the 134 participants who signed consent, 120 began study participation and were included in the baseline analysis.

Participant milestones

Participant milestones
Measure
Standard Practice
Participants received education regarding treatment consideration from their healthcare provider/team as per standard practice (usual care).
Standard Practice + Decision Aid
Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Overall Study
STARTED
60
60
Overall Study
COMPLETED
18
28
Overall Study
NOT COMPLETED
42
32

Reasons for withdrawal

Reasons for withdrawal
Measure
Standard Practice
Participants received education regarding treatment consideration from their healthcare provider/team as per standard practice (usual care).
Standard Practice + Decision Aid
Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Overall Study
Lost to Follow-up
42
32

Baseline Characteristics

Decision Aid for Therapeutic Options In Sickle Cell Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Standard Practice
n=60 Participants
Participants received education regarding treatment consideration from their healthcare provider/team as per standard practice (usual care).
Standard Practice + Decision Aid
n=60 Participants
Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Total
n=120 Participants
Total of all reporting groups
Age, Categorical
<=18 years
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
60 Participants
n=5 Participants
60 Participants
n=7 Participants
120 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Sex: Female, Male
Female
46 Participants
n=5 Participants
45 Participants
n=7 Participants
91 Participants
n=5 Participants
Sex: Female, Male
Male
14 Participants
n=5 Participants
15 Participants
n=7 Participants
29 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
60 Participants
n=5 Participants
60 Participants
n=7 Participants
120 Participants
n=5 Participants
Race (NIH/OMB)
White
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Region of Enrollment
United States
60 Participants
n=5 Participants
60 Participants
n=7 Participants
120 Participants
n=5 Participants

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure
Standard Practice + Decision Aid
Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Standard Practice and Standard Practice + Decision Aid Groups
n=106 Participants
Participants received standard of care teaching and discussion. Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Acceptability of Decision Aid Education Assessed by the Acceptability Survey
Research
3 units on a scale
Standard Deviation 1.1
Acceptability of Decision Aid Education Assessed by the Acceptability Survey
Treatment Options
3 units on a scale
Standard Deviation 1.1
Acceptability of Decision Aid Education Assessed by the Acceptability Survey
Health Impact of Sickle Cell Disease
3 units on a scale
Standard Deviation .86
Acceptability of Decision Aid Education Assessed by the Acceptability Survey
Risk Factors
3 units on a scale
Standard Deviation .97
Acceptability of Decision Aid Education Assessed by the Acceptability Survey
Evidence Supporting Self -Care
3 units on a scale
Standard Deviation 1.1
Acceptability of Decision Aid Education Assessed by the Acceptability Survey
Hydroxyurea/BMT/CBT
3 units on a scale
Standard Deviation 1.0
Acceptability of Decision Aid Education Assessed by the Acceptability Survey
Evidence About Hydroxyurea/BMT/CBT
3 units on a scale
Standard Deviation 1.1
Acceptability of Decision Aid Education Assessed by the Acceptability Survey
Stories About Others
3 units on a scale
Standard Deviation .64

SECONDARY outcome

Timeframe: Month 3, Month 6

Population: Analysis was completed for participants who completed the scale at month 3 and month 6 visits.

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.

Outcome measures

Outcome measures
Measure
Standard Practice + Decision Aid
n=16 Participants
Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Standard Practice and Standard Practice + Decision Aid Groups
n=11 Participants
Participants received standard of care teaching and discussion. Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Mean Decisional Self-Efficacy Scale Score
Month 3
75.31 units on a scale
Standard Deviation 22.1
59.29 units on a scale
Standard Deviation 22.4
Mean Decisional Self-Efficacy Scale Score
Month 6
84.84 units on a scale
Standard Deviation 16.39
71.36 units on a scale
Standard Deviation 29.97

SECONDARY outcome

Timeframe: Baseline, Month 3

Population: Analysis was completed for participants who completed the scale at both baseline and month 3 visits.

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).

Outcome measures

Outcome measures
Measure
Standard Practice + Decision Aid
n=22 Participants
Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Standard Practice and Standard Practice + Decision Aid Groups
n=20 Participants
Participants received standard of care teaching and discussion. Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Mean Difference in Decisional Conflict Scale Scores
Total Score
4.8 units on a scale
Interval -15.6 to 6.0
1.0 units on a scale
Interval -12.6 to 14.7
Mean Difference in Decisional Conflict Scale Scores
Uncertainty
1.9 units on a scale
Interval -9.4 to 13.2
.7 units on a scale
Interval -10.4 to 21.8
Mean Difference in Decisional Conflict Scale Scores
Informed
-12.1 units on a scale
Interval -23.3 to -1.0
-1.3 units on a scale
Interval -19.2 to 16.5
Mean Difference in Decisional Conflict Scale Scores
Values Clarity
-8.0 units on a scale
Interval -21.4 to 5.5
-.001 units on a scale
Interval -16.0 to 16.0
Mean Difference in Decisional Conflict Scale Scores
Support
4.6 units on a scale
Interval -17.7 to 8.6
-.4 units on a scale
Interval -16.3 to 15.4
Mean Difference in Decisional Conflict Scale Scores
Effective Decision
2.3 units on a scale
Interval -14.2 to 9.7
1.1 units on a scale
Interval -10.8 to 13.0

SECONDARY outcome

Timeframe: Baseline, Month 6

Population: Analysis was completed for participants who completed the scale at both baseline and month 6 visits.

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).

Outcome measures

Outcome measures
Measure
Standard Practice + Decision Aid
n=20 Participants
Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Standard Practice and Standard Practice + Decision Aid Groups
n=16 Participants
Participants received standard of care teaching and discussion. Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Mean Difference in Decisional Conflict Scale Scores
Total Score
-5.0 units on a scale
Interval -22.4 to 12.4
4.6 units on a scale
Interval -12.0 to 21.1
Mean Difference in Decisional Conflict Scale Scores
Uncertainty
.8 units on a scale
Interval -16.2 to 14.6
7.2 units on a scale
Interval -10.5 to 25.0
Mean Difference in Decisional Conflict Scale Scores
Informed
10.0 units on a scale
Interval -32.2 to 12.2
1.7 units on a scale
Interval -18.7 to 22.0
Mean Difference in Decisional Conflict Scale Scores
Values Clarity
-11.7 units on a scale
Interval -33.1 to 9.8
6.7 units on a scale
Interval -9.5 to 22.9
Mean Difference in Decisional Conflict Scale Scores
Support
1.3 units on a scale
Interval -18.1 to 15.6
.6 units on a scale
Interval -18.2 to 19.4
Mean Difference in Decisional Conflict Scale Scores
Effective Decision
-2.4 units on a scale
Interval -19.7 to 14.9
6.4 units on a scale
Interval -11.2 to 24.0

SECONDARY outcome

Timeframe: Post Visit 1 (Up to 2 Weeks)

Population: The analysis includes all participants who completed the survey.

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.

Outcome measures

Outcome measures
Measure
Standard Practice + Decision Aid
n=22 Participants
Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Standard Practice and Standard Practice + Decision Aid Groups
n=20 Participants
Participants received standard of care teaching and discussion. Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Mean Values Survey Score
10 units on a scale
Standard Deviation 1.7
10.2 units on a scale
Standard Deviation 2

SECONDARY outcome

Timeframe: Month 3, Month 6

Population: The analysis includes all participants who completed the survey.

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.

Outcome measures

Outcome measures
Measure
Standard Practice + Decision Aid
n=16 Participants
Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Standard Practice and Standard Practice + Decision Aid Groups
n=11 Participants
Participants received standard of care teaching and discussion. Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Mean Change in Preparation for Decision Making Scale Score
.38 units on a scale
Standard Deviation .74
.48 units on a scale
Standard Deviation .89

SECONDARY outcome

Timeframe: Baseline, Month 3, Month 6

Population: The analysis includes all participants who completed the survey.

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%.

Outcome measures

Outcome measures
Measure
Standard Practice + Decision Aid
n=39 Participants
Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Standard Practice and Standard Practice + Decision Aid Groups
n=38 Participants
Participants received standard of care teaching and discussion. Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Mean Knowledge Survey Scores
Baseline
39 units on a scale
Standard Deviation 49.83
38 units on a scale
Standard Deviation 49.87
Mean Knowledge Survey Scores
Month 3
23 units on a scale
Standard Deviation 52.90
19 units on a scale
Standard Deviation 52.90
Mean Knowledge Survey Scores
Month 6
22 units on a scale
Standard Deviation 55.54
46.79 units on a scale
Standard Deviation 51.18

SECONDARY outcome

Timeframe: Visit 3

Population: Number of participants who completed the scale.

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.

Outcome measures

Outcome measures
Measure
Standard Practice + Decision Aid
Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Standard Practice and Standard Practice + Decision Aid Groups
n=7 Participants
Participants received standard of care teaching and discussion. Participants received standard of care teaching and discussion in addition to web-based decision aid tool access.
Mean Decisional Regret Scale Score
27.5 units on a scale
Standard Deviation 23.2

Adverse Events

Standard Practice

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Standard Practice + Decision Aid

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Lakshmanan Krishnamurti, MD

Emory University

Phone: 4047270710

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place