Motivations, expectations,and decision making of sickle cell patients in clinical research

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Clinical trials testing potentially curative interventions for sickle cell disease such as gene therapy (GT) or peripheral blood stem cell transplantation (PBSCT) have created a novel opportunity for patients with sickle cell disease, but may pose risk of significant adverse events. As the development of these interventions create a new decision-making situation for persons with longstanding diseases, these trials raise questions about the expectations and decision-making process of patients considering and/or participating in them. This study explores the expectations, understanding, motivations, and decision-making of patients with sickle cell disease who have either chosen to enroll or declined to enroll in research studies testing experimental interventions. Patients who have decided to enroll or decline in a PBSCT or GT protocol for sickle cell will be invited for an interview. The interview will ask about their experience with sickle cell disease, how they found out about the intervention study and how they made the decision to or not to enroll, including their understanding, motivations, support systems, etc. We interviewed 26 patients, and published the results in Biology of Blood and Marrow Transplantation.