Clinical trial NCT03577691: Efficacy of a Decision Aid for Hydroxyurea in Sickle Cell Disease
Official title: Efficacy of a Decision Aid for Hydroxyurea(HU) in Sickle Cell Disease(SCD)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: NA
Conditions: Sickle Cell Disease
Keywords: Blood Disorders
Hydroxyurea (HU)
Decision Aid
Lead sponsor: Emory University
Sponsor class: OTHER
First posted: 2018-07-05
Enrollment: 2
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: FACTORIAL
Intervention model description: Prospective, randomized,controlled, unblinded study
Primary purpose: SUPPORTIVE_CARE

Brief summary: The trial is a prospective, randomized study to determine the efficacy of training for use of a web based Decision Aid for Hydroxyurea(HU) usage among subjects with Sickle Cell Disease(SCD). Subjects are randomized primarily either to using a web based decision aid with training versus without training. Subjects in each group will be further randomized to 1) subjects receiving pretest surveys; and 2) subjects that do not receive pretest surveys.

Detailed description: Hydroxyurea (HU) has been demonstrated to be efficacious in reducing complications such as vasocclusive pain crises and acute chest syndrome in children and adults with Sickle Cell Disease (SCD) and in improving survival in adults. It is also the only disease-modifying therapy and the only drug approved by the FDA for use in patients with SCD. However significant barriers remain in prescription and utilization of HU. Some of the barriers are under prescription and when prescribed, underutilization by patients. The majority of the reasons for underutilization by patients being fear of cancer or other side effects, concern about lack of efficacy, and unwillingness to take the medicine or come to clinic or pharmacy.

Decision aids are designed to help patients improve their knowledge of the options, achieve a more accurate perception of risk and benefits of treatment, participate actively in decision making, and make choices more in keeping with their values and preferences. Decision aids have been shown to be effective in providing greater knowledge; lower decisional conflict among the subjects. The web based decision aid is also likely to serve as a tool for clarification of patient values to themselves and to their healthcare providers. The web based decision aid, sickleoptions.org is a rather extensive and detailed website with information regarding sickle cell disease(SCD) complications and standard of care monitoring and management as well as the treatment options of hydroxyurea, Endari, chronic blood transfusion, and bone marrow transplant. It is necessary to assure user ability to navigate all of the elements of the site. The study wants to determine the efficacy of training for a web based Decision Aid for Hydroxyurea(HU) usage among subjects with Sickle Cell Disease(SCD) that are randomized to using a web based decision aid with training versus without training.

In the first phase of the study cognitive interviews will be conducted with 10-20 parents of patients with SCD or patients with SCD age \> 18 years for development of training to navigate website. Interviews will be conducted in 5-subject iterative cycles to identify ease of usability and issues regarding navigation and usability to develop a training guide for website.

After consent, subjects will be asked to complete demographic questionnaires. Subjects will be randomized to either receive training or to not receive training to use the website. All subjects will be asked to spend about 30mts navigating the website after consent. Those who are randomized to receive training will be trained to use it during this time. Those who do not receive training will be asked to navigate through the site on their own. They may be randomized to complete 4 question surveys during this time. Not everyone will need to complete these surveys. This will take about 15 minutes. They will then be contacted each week for the next 3 weeks with a reminder to use the website. At week 4 they will complete 5 surveys over the phone. They may be randomized to complete a telephone interview. This interview will take about 30 to 45 minutes and will be audio recorded for transcription and analysis. Subjects will then be contacted every 3 months for 1 year to see if they made a decision about whether or not to start hydroxyurea for themselves (\>18 yr-old subjects) or their child. These phone calls will take less than 5 minutes.

Eligibility criteria: Inclusion Criteria:

* Individuals with sickle cell disease ages 18 to 80 years, inclusive Or parents of children (ages 9 months to \<18 years) whose child has not started Hydroxyurea
* Patients who have made a past decision to not obtain treatment with Hydroxyurea or who have not obtained treatment with Hydroxyurea in past 12 months. Or parents of children who have made a past decision to not obtain treatment with Hydroxyurea or who have not obtained treatment with Hydroxyurea in the past 12 months.
* Patients or parents of children who's doctor identifies the patient/child as someone who should be on Hydroxyurea
* All participants will be able to comprehend English
* Subjects will have access to the internet from iPad, smart phone, or personal computer
* For the cognitive interviews participants will not be eligible to consider hydroxyurea as a treatment option

Exclusion Criteria:

* Patient who has already made a decision to begin and has started Hydroxyurea. Or parent who has already made a decision for their child to begin and the child has started Hydroxyurea.
* Participated in prior Decision Aid study

Primary outcomes:
measure: Change in Knowledge Survey score to estimate the impact of use of the Decision Aid with training versus without training in improving knowledge regarding HydroxyUrea for Sickle Cell Disease
timeFrame: Baseline and 4 week follow up
Secondary outcomes:
measure: Change in decisional conflict scale to estimate the impact of use of the Decision Aid with training versus without training aid on decisional conflict
timeFrame: Baseline and 4 week follow up
measure: Change in decision self efficacy scale to estimate the impact of use of the Decision Aid with training versus without training aid on decisional conflict
timeFrame: Baseline and 4 week follow up
measure: Change in Preparation for Decision Making Scale to estimate the impact of use of the Decision Aid with training versus without training aid on decisional conflict
timeFrame: Baseline and 4 week follow up
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Grady Health System
state: Georgia
city: Atlanta
country: United States
facility: Hughes Spalding Children's Hospital
state: Georgia
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
Study officials:
affiliation: Emory University
name: Lakshmanan Krishnamurti, MD
role: PRINCIPAL_INVESTIGATOR
References: