Decision Aid for Therapeutic Options In Sickle Cell Disease

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.

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.