Individuals living with severe sickle cell disease (SCD) are highly interested in new, potentially curative gene therapy treatments and are willing to accept associated risks for a chance at a cure, according to a study published in Blood Advances.
Amidst several gene therapies pending U.S. Food and Drug Administration (FDA) approval, Dr. Juan Marcos Gonzalez and his team initiated a study to understand patient views on these novel treatments. They used a discrete-choice experiment survey, which presented participants with pairs of hypothetical gene therapy treatments along with a ‘no gene therapy’ option. The gene therapy choices were characterized by their potential to eliminate SCD symptoms, extend life expectancy, and their associated risks, including treatment-related mortality, infertility, and increased cancer risk.
Across three U.S. clinical sites and with the support of Cayenne Wellness, a patient-driven organization, 174 adult patients and 109 parents of children living with SCD completed the survey. Most survey respondents were willing to choose gene therapy over alternatives. However, when gene therapy posed a 10% or greater risk of death, adults with mild symptoms required a higher likelihood of eliminating SCD symptoms (around 67% on average) than parents of children (around 8%) to ultimately choose gene therapy. In contrast, adults experiencing moderate symptoms required lower success rates. To accept gene therapies posing 10% or 30% mortality risks, these patients required 34% and 37% chance of having their SCD symptoms fully resolved, respectively.
Both adults living with SCD and parents of children with SCD most often opted for gene therapies offering higher chances of eliminating SCD symptoms and giving a longer life expectancy, and less often opted for gene therapies with higher chances of death, controlling for other factors.
