Motivations, Expectations, and Decision-making of Sickle Cell Patients in Clinical Research

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, for whom standard therapies can only manage but not cure their conditions. But some of these experimental interventions 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. Given the longstanding debate in the bioethics literature about whether patients expressions of desire for benefit are (1) evidence of false hopes or poor understanding of risks and benefits, or, instead (2) expressions of natural optimism compatible with valid informed consent, these patients present an opportunity to learn about how they make their participation decisions which in turn could inform this longstanding debate.

This study aims to explore the expectations, understanding, motivations, and decision-making of patients with sickle cell disease who have either chosen or declined to enroll in research studies testing experimental interventions. The aim is to better understand how these patients understand the research study, assess risks and benefits at the time of enrollment, make decisions, and react to their health outcomes. As faith and religion are known to play an important part in the lives of persons with sickle cell disease, we will also explore the role of religion and faith in sickle cell patients decision-making and retrospective perspective on their decision.

Primary Hypothesis:

This is a descriptive, explorative study. It may generate hypotheses for future studies.

Purpose of the Study Protocol:

To describe how patients with sickle cell disease understand and make decisions about participating in gene therapy (GT) or peripheral blood stem cell transplantation (PBSCT) clinical trials to shed light about the ethically salient issues regarding enrollment and participation in high- risk, high-reward clinical trials. This information may inform practices around the informed consent process and help researchers better understand the role of family and religion/faith in clinical research decision making.

• INCLUSION CRITERIA:

Our inclusion criteria for study subjects are:

• Adult (18+ years and older)
• Sickle cell disease patients who are enrolled in at least one of the following sickle cell disease protocols at the National Institutes of Health, National Heart, Lung, and Blood Disease:

• Screening study: 08-H-0156
• Natural history protocol: 04-H-0161
• PBSCT: 09-H-0225, 17-H-0069, 14-H-0077, 03-H-0170
• GT: 14-H-0155
• Have made a decision regarding participation in one of the GT or PBSCT protocols (e.g. someone could still be enrolled in Screening study, have made a decision, but not yet enrolled in or have declined enrollment in the GT or PBSCT studies)
• Study subjects will be recruited in two groups: 1) pre-transplant or pre-initiation of gene therapy, including both those who decide to enroll and those who declined to enroll; and 2) post-transplantation or post-GT, including those who had an unsuccessful and those who had a successful transplantation or response to GT.

Exclusion criteria

Study subjects will be excluded if they:

• Lack cognitive capacity
• Are not English speaking
• Have not made a decision about participation in one of the GT or PBSCT protocols for which they are eligible