Efficacy of a Decision Aid for Hydroxyurea in Sickle Cell Disease

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.

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