Decision Aid for Therapeutic Options In Sickle Cell Disease

Sickle Cell Disease Clinical Trial

Official title:

Comparative Effectiveness of a Decision Aid for Therapeutic Options in Sickle Cell Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02326597
• Other study ID #: IRB00076096a
• Status: Completed
• Phase: N/A
• Start date: January 2015
• Est. completion date: April 17, 2017

Study information

• Verified date: December 2017
• Source: Emory University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 134
• Est. completion date: April 17, 2017
• Est. primary completion date: April 17, 2017
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 8 Years to 80 Years

Eligibility

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.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• beta-Thalassemia
• Hemoglobin Beta Thalassemia
• Hemoglobin SC
• Hemoglobin SC Disease
• Hemoglobin SS
• Sickle Cell Anemia
• Sickle Cell Disease
• Thalassemia

Intervention

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

Locations

Country	Name	City	State
United States	Emory University	Atlanta	Georgia

Sponsors (2)

Lead Sponsor	Collaborator
Emory University	Patient-Centered Outcomes Research Institute

Country where clinical trial is conducted

United States, 

References & Publications (8)

Aloe A, Krishnamurti L, Kladny B. Testing of collegiate athletes for sickle cell trait: what we, as genetic counselors should know. J Genet Couns. 2011 Aug;20(4):337-40. doi: 10.1007/s10897-011-9366-9. Epub 2011 Apr 19. — View Citation

Dovey S, Krishnamurti L, Sanfilippo J, Gunawardena S, Mclendon P, Campbell M, Alway S, Efymow B, Gracia C. Oocyte cryopreservation in a patient with sickle cell disease prior to hematopoietic stem cell transplantation: first report. J Assist Reprod Genet. 2012 Mar;29(3):265-9. doi: 10.1007/s10815-011-9698-2. Epub 2012 Jan 5. — View Citation

Kladny B, Williams A, Gupta A, Gettig EA, Krishnamurti L. Genetic counseling following the detection of hemoglobinopathy trait on the newborn screen is well received, improves knowledge, and relieves anxiety. Genet Med. 2011 Jul;13(7):658-61. doi: 10.1097/GIM.0b013e31821435f7. — View Citation

Krishnamurti L, Kharbanda S, Biernacki MA, Zhang W, Baker KS, Wagner JE, Wu CJ. Stable long-term donor engraftment following reduced-intensity hematopoietic cell transplantation for sickle cell disease. Biol Blood Marrow Transplant. 2008 Nov;14(11):1270-8. doi: 10.1016/j.bbmt.2008.08.016. — View Citation

Long KA, Thomas SB, Grubs RE, Gettig EA, Krishnamurti L. Attitudes and beliefs of African-Americans toward genetics, genetic testing, and sickle cell disease education and awareness. J Genet Couns. 2011 Dec;20(6):572-92. doi: 10.1007/s10897-011-9388-3. Epub 2011 Jul 12. — View Citation

Nouraie M, Lee JS, Zhang Y, Kanias T, Zhao X, Xiong Z, Oriss TB, Zeng Q, Kato GJ, Gibbs JS, Hildesheim ME, Sachdev V, Barst RJ, Machado RF, Hassell KL, Little JA, Schraufnagel DE, Krishnamurti L, Novelli E, Girgis RE, Morris CR, Rosenzweig EB, Badesch DB, Lanzkron S, Castro OL, Goldsmith JC, Gordeuk VR, Gladwin MT; Walk-PHASST Investigators and Patients. The relationship between the severity of hemolysis, clinical manifestations and risk of death in 415 patients with sickle cell anemia in the US and Europe. Haematologica. 2013 Mar;98(3):464-72. doi: 10.3324/haematol.2012.068965. Epub 2012 Sep 14. — View Citation

Rao VK, Price S, Perkins K, Aldridge P, Tretler J, Davis J, Dale JK, Gill F, Hartman KR, Stork LC, Gnarra DJ, Krishnamurti L, Newburger PE, Puck J, Fleisher T. Use of rituximab for refractory cytopenias associated with autoimmune lymphoproliferative syndrome (ALPS). Pediatr Blood Cancer. 2009 Jul;52(7):847-52. doi: 10.1002/pbc.21965. — View Citation

Wu CJ, Gladwin M, Tisdale J, Hsieh M, Law T, Biernacki M, Rogers S, Wang X, Walters M, Zahrieh D, Antin JH, Ritz J, Krishnamurti L. Mixed haematopoietic chimerism for sickle cell disease prevents intravascular haemolysis. Br J Haematol. 2007 Nov;139(3):504-7. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
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.	Post Visit 1 (Up to 2 Weeks)
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.	Month 3, Month 6
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).	Baseline, Month 3
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).	Baseline, Month 6
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.	Post Visit 1 (Up to 2 Weeks)
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.	Month 3, Month 6
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%.	Baseline, Month 3, Month 6
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.	Visit 3