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Decision Aid for Therapeutic Options In Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT02326597
Recruitment Status : Completed
First Posted : December 29, 2014
Results First Posted : October 8, 2018
Last Update Posted : October 8, 2018
Sponsor:
Collaborator:
Patient-Centered Outcomes Research Institute
Information provided by (Responsible Party):
Lakshmanan Krishnamurti, Emory University

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.


Condition or disease Intervention/treatment Phase
Sickle Cell Disease Sickle Cell Anemia Hemoglobin SS Hemoglobin SC Hemoglobin Beta Thalassemia Other: Decision Aid Tool Other: Standard Practice Not Applicable

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.


Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 134 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary Purpose: Health Services Research
Official Title: Comparative Effectiveness of a Decision Aid for Therapeutic Options in Sickle Cell Disease
Actual Study Start Date : January 2015
Actual Primary Completion Date : April 17, 2017
Actual Study Completion Date : April 17, 2017


Arm Intervention/treatment
Active Comparator: Standard Practice
Participants will receive education regarding treatment consideration from their healthcare provider/team as per standard practice (usual care).
Other: Standard Practice
Standard of care teaching and discussion regarding treatment options given by patient's healthcare provider.

Experimental: Standard Practice + Decision Aid
Participants will receive standard of care teaching and discussion in addition to web-based decision aid tool access.
Other: Decision 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.

Other: Standard Practice
Standard of care teaching and discussion regarding treatment options given by patient's healthcare provider.




Primary Outcome Measures :
  1. Acceptability of Decision Aid Education Assessed by the Acceptability Survey [ Time Frame: 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 Outcome Measures :
  1. Mean Decisional Self-Efficacy Scale Score [ Time Frame: 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.

  2. Mean Difference in Decisional Conflict Scale Scores [ Time Frame: 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).

  3. Mean Difference in Decisional Conflict Scale Scores [ Time Frame: Baseline, Month 6 ]
    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).

  4. Mean Values Survey Score [ Time Frame: 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.

  5. Mean Change in Preparation for Decision Making Scale Score [ Time Frame: 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.

  6. Mean Knowledge Survey Scores [ Time Frame: 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%.

  7. Mean Decisional Regret Scale Score [ Time Frame: 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   8 Years to 80 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02326597


Locations
United States, Georgia
Emory University
Atlanta, Georgia, United States, 30322
Sponsors and Collaborators
Emory University
Patient-Centered Outcomes Research Institute
Investigators
Principal Investigator: Lakshmanan Krishnamurti, MD Emory University
  Study Documents (Full-Text)

Documents provided by Lakshmanan Krishnamurti, Emory University:

Publications:

Responsible Party: Lakshmanan Krishnamurti, Professor, Emory University
ClinicalTrials.gov Identifier: NCT02326597     History of Changes
Other Study ID Numbers: IRB00076096a
First Posted: December 29, 2014    Key Record Dates
Results First Posted: October 8, 2018
Last Update Posted: October 8, 2018
Last Verified: December 2017

Keywords provided by Lakshmanan Krishnamurti, Emory University:
Hydroxyurea
Bone marrow transplant
Chronic blood transfusion
Treatment options for sickle cell disease
Decision making

Additional relevant MeSH terms:
Anemia, Sickle Cell
Thalassemia
beta-Thalassemia
Hemoglobin SC Disease
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn