This paper explores the importance of hope as a determining factor for patients to participate in first-in-human trials for synthetic biology therapies. This paper focuses on different aspects of hope in the context of human health and well-being and explores the varieties of hope expressed by patients. The research findings are based on interview data collected from stable gout and diabetes patients. Three concepts of hope have emerged from the interviews: hope as certainty ; hope as reflective uncertainty ; hope (...) as self-therapy. The purpose of the paper is twofold. First, it aims to underline the significance of hope in patients’ medical decision-making, as well as the beneficence of hope for patients’ well-being, and for progress in research. Second, it shows how philosophical investigations—in particular Descartes—explore the phenomenon of hope and provide medical empirical research with profitable insights and tools. (shrink)

BackgroundBeta thalassemia major is a severe inherited form of hemolytic anemia that results from ineffective erythropoiesis. Allogenic hematopoietic stem cell transplantation (HSCT) remains the only potentially curative therapy. Unfortunately, the subgroup of adult thalassemia patients with hepatomegaly, portal fibrosis and a history of irregular iron chelation have an elevated risk for transplantation-related mortality that is currently estimated to be about 29 percent.DiscussionThalassemia patients may be faced with a difficult choice: they can either continue conventional transfusion and iron chelation therapy or (...) accept the high mortality risk of HSCT in the hope of obtaining complete recovery.Throughout the decision making process, every effort should be made to sustain and enhance autonomous choice. The concept of conscious consent becomes particularly important. The patient must be made fully aware of the favourable and adverse outcomes of HSCT. Although it is the physician's duty to illustrate the possibility of completely restoring health, considerable emphasis should be put on the adverse effects of the procedure. The physician also needs to decide whether the patient is eligible for HSCT according to the "rule of descending order". The patient must be given full details on self-care and fundamental lifestyle changes and be fully aware that he/she will be partly responsible for the outcome.SummaryOnly if all the aforesaid conditions are satisfied can it be considered reasonable to propose unrelated HSCT as a potential cure for high risk thalassemia patients. (shrink)