Strengthening advanced therapy for sickle cell disease in Africa: experience from sickle cell disease centre in Dar es Salaam, Tanzania.

Despite progress in healthcare services for individuals living with sickle cell disease (SCD) in Africa, substantial gaps remain in advanced treatments for SCD. To help address this burden, Tanzania has established one of the largest single-centre SCD programmes in the world and developed an advanced therapy programme for SCD focused on patient engagement and advocacy, clinical activities involving exchange blood transfusion (ExBT) and haematopoietic stem cell transplant (HSCT), gene therapy (GT) preparedness, and enabling partnerships. This report describes the programme's genesis, structure and progress achieved. Patient engagement camps and patient-focused workshops conducted since early 2021 have involved more than 150 patients, family caregivers and healthcare providers. A patient registry was established by screening 1500 patients eligible for advanced therapies with 157 identified to benefit from advanced treatments for SCD. Out of which 22 patients received ExBT, and human leucocyte antigen typing was conducted on 127 individuals to establish a registry of family members with potential to be HSCT donors. Target product profiles were devised for minimum and optimum criteria of GT products to guide drug discovery and development efforts, and qualitative research was conducted to investigate factors anticipated to influence successful adoption of GTs for SCD in Africa. The programme's multifaceted components have been enabled by institutional networks and collaborations established at national, regional and global levels. The programme presented opportunities to deliver cost-effective advanced treatment and curative options for SCD in Tanzania and lessons learnt may be applicable to inform similar efforts in other African regions where SCD is highly endemic.