Motivations and Decision-Making of Adult Sickle Cell Patients in High-Risk Clinical Research

Abstract

Potentially curative but high-risk trials of gene therapy or stem cell transplantation (PBSCT) for sickle cell disease (SCD) pose new opportunities for adults with SCD, many of whom experience significant disease burden and complications with few treatment options, as well as stigma and disparities in care. We explored motivations and decision-making processes of enrollees and decliners of such trials. Semistructured interviews were conducted with a purposive sample of 20 enrollees and 6 decliners. Interviews explored participants' SCD experiences, motivations, and decision-making about trial participation, understanding of research-related information, and retrospective reflections. Interviews were analyzed with content analysis. Most identified the purpose of research, risks, and uncertainties of participation. Both enrollees and decliners described deliberative weighing of study risks and potential benefits (especially the prospect of a cure), with heavy factoring of their SCD status, experiences, and desire for a better life. Despite the influence of spirituality/religion and support of family and friends, all described the decision about participation as their own. In some patients, the primary outcome status defined by the trial did not match the patients' perceived outcomes. Patients with negative experiences expressed a desire for greater emphasis on risks and possible outcomes during informed consent. This cohort of adults with SCD were thoughtfully deliberative in their decisions about gene therapy or PBSCT trials. Future participants' decisionmaking may be enhanced by emphasizing that "successful" scientific outcomes can still involve complications or symptoms and be facilitated by referrals to former research participants and anticipatory discussions.Published by Elsevier Inc. on behalf of the American Society for Transplantation and Cellular Therapy.

A experiência de adoecimento falciforme pelas lentes qualitativas

“…A sexta dimensão [72][73][74][75][76] que emerge no acervo é o da participação dos conviventes com a DF em processos de decisão, seja na participação em pesquisas clínicas seja na comunicação com os profissionais de saúde na escolha de tratamentos terapêuticos. Destacamos aqui como a participação tem um papel muito relevante de construção da autoestima frente ao adoecimento.…”

The sickle cell illness experience under the qualitative lens

“…The sixth dimension [72][73][74][75][76] that emerges in the collection is the participation of those living with SCD in decision processes, either in participating in clinical research or communicating with health professionals to choose treatments. We highlight how participation is crucial in building self-esteem in the face of illness.…”

Genetic therapies for the first molecular disease

“…Translating scientific advances into SCD patient care is challenging. Participation in high-risk, potentially curative clinical trials can be influenced by patientperceived disease burden, expected benefits, fear of toxicities, and socio-ecological factors including anticipated stress, religious beliefs, and support systems (24)(25)(26). Other barriers include lack of appropriate educational material, therapy-associated costs, and limited access to longitudinal care.…”

“…Given the uncertainties of high-risk therapeutic clinical trials, it may be valuable to conduct psychosocial assessments evaluating the emotional function, coping ability, and social support stability of potential participants to facilitate informed consent and better manage outcome expectations. Offering coping strategies and opportunities for clinical trial participants to explore potentially unexpected or less favorable outcomes enhances the shared decision-making model (26).…”