Stem cell transplant for sickle cell disease

Thesis (M.A.)--Boston UniversityBackground: Sickle cell disease (SCD) is the most common inherited blood disorder in the United States. As SCD can cause significant morbidity and decrease in life expectancy, further research on curative options is of great interest. Hematopoietic stem cell transplant (HSCT) is the only treatment option offering a chance of cure, but the risks of treatment are not negligible. Because the outcomes of HSCT are best when the procedure is performed at a younger age, understanding what parents know about transplant, their opinion on this option and the risks they are willing to take to achieve a cure is of great value. As sickle cell disease has changed in the United States from a life-threatening condition of childhood to a chronic condition with most of the burden of morbidity and mortality shifted towards adulthood, it is necessary for parents to be fully aware of long term risks and educated on all therapeutic options, so the optimal decision can be made. Objectives: (i) To learn about parents’ recollection and pursuit of further information after undergoing an educational session on risks and benefits of HSCT. (ii) To learn about their worries about transplant and the highest mortality and infertility risks they are willing to accept in order to achieve a cure for their child. (iii) To learn about parents’ readiness to proceed to transplant based on a hypothetical scenario. [TRUNCATED