Optimizing hematopoietic stem cell transplantation outcomes

Hematopoietic stem cell transplantation (HSCT) is an important treatment option for many hematologic diseases, offering the possibility of cure or long-term remission. Over the years, several major barriers have been overcome, and improvements in donor availability, conditioning regimens, Graft-versus-Host disease (GvHD) prophylaxis, and supportive care have led to better outcomes. Despite these advances, HSCT (especially allogeneic) remains a complex treatment with significant risk of complications, leading to transplantation-related morbidity and mortality. Further progress is needed, both in the transplant approach and in the supportive care surrounding it. Many of the current challenges are more subtle than before and require careful evaluation of long-standing practices. This thesis focuses on these areas, addressing established treatment strategies that may benefit from refinement and stronger evidence to improve patient care and transplantation outcomes. Part I of this thesis focuses on clinical studies in malignant hematologic diseases, addressing key aspects of both autologous and allogeneic HSCT. Part II centers on developments in transplantation of adults with sickle cell disease.