decision aid for therapeutic options in sickle cell disease

Conditions: Sickle Cell Disease; Sickle Cell Anemia; Hemoglobin SS; Hemoglobin SC; Hemoglobin Beta Thalassemia

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.

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.

Keywords: hydroxyurea bone marrow transplant chronic blood transfusion treatment options for sickle cell disease decision making