Clinical trial NCT02326597: Decision Aid for Therapeutic Options In Sickle Cell Disease
Official title: Comparative Effectiveness of a Decision Aid for Therapeutic Options in Sickle Cell Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
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
Lead sponsor: Emory University
Sponsor class: OTHER
Collaborators: name: Patient-Centered Outcomes Research Institute
First posted: 2014-12-29
Enrollment: 134
Sex: ALL
Minimum age (years): 8
Maximum age (years): 80
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: HEALTH_SERVICES_RESEARCH

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.

Eligibility 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.

Primary outcomes:
measure: Acceptability of Decision Aid Education Assessed by the Acceptability Survey
timeFrame: Post Visit 1 (Up to 2 Weeks)
Secondary outcomes:
measure: Mean Decisional Self-Efficacy Scale Score
timeFrame: Month 3, Month 6
measure: Mean Difference in Decisional Conflict Scale Scores
timeFrame: Baseline, Month 3
measure: Mean Difference in Decisional Conflict Scale Scores
timeFrame: Baseline, Month 6
measure: Mean Values Survey Score
timeFrame: Post Visit 1 (Up to 2 Weeks)
measure: Mean Change in Preparation for Decision Making Scale Score
timeFrame: Month 3, Month 6
measure: Mean Knowledge Survey Scores
timeFrame: Baseline, Month 3, Month 6
measure: Mean Decisional Regret Scale Score
timeFrame: Visit 3
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Emory University
state: Georgia
Study officials:
affiliation: Emory University
name: Lakshmanan Krishnamurti, MD
role: PRINCIPAL_INVESTIGATOR
References:
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. doi: 10.1111/j.1365-2141.2007.06803.x. No abstract available.
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.
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.
citation: 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.
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.
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.
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.
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.
citation: 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.