The landscape of therapy in patients with SCD has recently moved toward more personalized therapeutic approaches. Promising preliminary data were obtained with some disease-modifying agents and gene therapies became available and promising for curing patients. However, the high cost remains a major limiting factor for these new therapies, which are far from being used in all patients, even in the more developed countries. Hydroxyurea is still regarded as the best disease-modifying treatment for SCD and allogeneic hematopoietic stem cell transplantation as the standard 'curative' therapy. Continued research is still warranted to sustain therapeutic advancements and development in SCD.
Giovanna Cannas (Mon,) studied this question.