SCD is increasingly recognized as a chronic inflammatory condition, highlighting the need for deeper insight into phenotype and function of (adaptive) immune cells. At the same time, as Haplo-HSCT is increasingly used in adults, critical questions remain regarding its impact on immune dysregulation in SCD, as well as clinical challenges such as infections, mixed chimerism, and overt graft
failure, particularly when using bone marrow as the stem cell source.
Research questions
1.) Does Haplo-HSCT restore the immunophenotype and function of PBMCs in adults with SCD?
2.) How and when do PBMCs reconstitute following Haplo-HSCT, and are specific subsets linked to transplantation outcomes, such as infections or mixed chimerism?
3.) What are the differences in immune reconstitution and transplantation outcomes between bone marrow and peripheral blood as stem cell source? Bone marrow is currently standard of care, though often limited by insufficient CD34+ cell yields. Peripheral blood stem cells, which provide larger counts of CD34+ cells, may enhance engraftment and improve immune reconstitution, but are associated with increased risk of GvHD?