Decision Aid for Therapeutic Options In Sickle Cell Disease

NCT02326597 | Completed Results

• 1 other identifier: IRB00076096a
• Study Type: interventional
• Target: 134
• Locations: 1 country
• Sites: 1

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.

Conditions

Sickle Cell Disease; Sickle Cell Anemia; Hemoglobin SS; Hemoglobin SC; Hemoglobin Beta Thalassemia

Keywords

Hydroxyurea; Bone marrow transplant; Chronic blood transfusion; Treatment options for sickle cell disease; Decision making

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (7)

• Mean Decisional Self-Efficacy Scale Score

  Month 3, Month 6

• Mean Difference in Decisional Conflict Scale Scores

  Baseline, Month 3

• Mean Difference in Decisional Conflict Scale Scores

  Baseline, Month 6

• Mean Values Survey Score

  Post Visit 1 (Up to 2 Weeks)

• Mean Change in Preparation for Decision Making Scale Score

  Month 3, Month 6

Study Arms (2)

Standard Practice

ACTIVE COMPARATOR

Participants will receive education regarding treatment consideration from their healthcare provider/team as per standard practice (usual care).

Other: Standard Practice

Standard Practice + Decision Aid

EXPERIMENTAL

Participants will receive standard of care teaching and discussion in addition to web-based decision aid tool access.

Other: Decision Aid Tool; Other: Standard Practice

Interventions

Decision Aid Tool OTHER

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 + Decision Aid

Standard Practice OTHER

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

Standard Practice; Standard Practice + Decision Aid

Eligibility Criteria

• Age: 8 Years - 80 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Emory University: lead
• Patient-Centered Outcomes Research Institute: collaborator

Study Sites (1)

Emory University

Atlanta, Georgia, 30322, United States

Location

Related Publications (9)

• 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. doi: 10.1111/j.1365-2141.2007.06803.x. No abstract available.

  PMID: 17910640 BACKGROUND

• 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.

  PMID: 18940682 BACKGROUND

• 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.

  PMID: 19214977 BACKGROUND

• 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.

  PMID: 21503822 BACKGROUND

• 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.

  PMID: 22219083 BACKGROUND

• 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.

  PMID: 21546841 BACKGROUND

• 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.

  PMID: 21748660 BACKGROUND

• 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.

  PMID: 22983573 BACKGROUND

• Krishnamurti L, Ross D, Sinha C, Leong T, Bakshi N, Mittal N, Veludhandi D, Pham AP, Taneja A, Gupta K, Nwanze J, Matthews AM, Joshi S, Vazquez Olivieri V, Arjunan S, Okonkwo I, Lukombo I, Lane P, Bakshi N, Loewenstein G. Comparative Effectiveness of a Web-Based Patient Decision Aid for Therapeutic Options for Sickle Cell Disease: Randomized Controlled Trial. J Med Internet Res. 2019 Dec 4;21(12):e14462. doi: 10.2196/14462.

  PMID: 31799940 DERIVED

MeSH Terms

Conditions

Anemia, Sickle Cell; Hemoglobin SC Disease

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Lakshmanan Krishnamurti, MD
• Organization: Emory University

lkrishn@emory.edu

4047270710

Study Officials

• Lakshmanan Krishnamurti, MD

  Emory University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: HEALTH SERVICES RESEARCH
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: December 18, 2014
• First Posted: December 29, 2014
• Study Start: January 1, 2015
• Primary Completion: April 17, 2017
• Study Completion: April 17, 2017
• Last Updated: October 9, 2018
• Results First Posted: October 9, 2018

Record last verified: 2017-12

Locations

US (1)