Decision Aid for Therapeutic Options In Sickle Cell Disease
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.

 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.
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.