ABSTRACT
Globally, sickle cell disease (SCD) accounts for the highest burden of morbidity and mortality amongst all genetic disorders, with 80% of the affected population residing in sub-Saharan Africa (SSA). Being a chronic disease, marked by acute complications and extremely variable course, the trajectory of the disease in a given individual remains unpredictable. This problem is compounded by the lack of any curative option other than an allogeneic haematopoietic cell transplantation (Allo-HCT). Despite the slow pace of development in the field of Allo-HCT for SCD in Europe and the USA, compared to other diseases, long-term SCD-free survival in children who have undergone an Allo-HCT in the last two decades is in excess of 90%, when carried out from a human leukocyte antigen (HLA)-matched family donor (MFD).
This begs the question that despite the enormity of the success of HCT in SCD, why is it not offered more widely across the globe, particularly in the most affected regions of the world. We shall discuss the current status of Allo-HCT in SCD and how this can be scaled up to reach the most affected regions in the world.
