Table 2.

Therapeutic options for SCA with potential relevance for sub-Saharan Africa

TreatmentAdvantages and indicationsDisadvantages and challenges
Erythrocyte transfusions• Treatment of severe anemia due to splenic sequestration, parvovirus infection, or malaria• Lack of sufficient blood donors
• Additional oxygen-carrying capacity for life-threatening acute vaso-occlusion and organ damage• Infection transmission (HIV, hepatitis B and C, syphilis)
• Effective treatment option for stroke and other neurologic complications• Erythrocyte alloimmunization
• Inability to prepare blood components
• Eventual need for iron chelation
Hydroxyurea• Reduction of acute vaso-occlusive complications (pain, acute chest syndrome)• Limited drug availability
• Oral administration• High cost relative to daily wages
• Once-daily dosing• Optimal dosing not yet determined
• Documented laboratory and clinical efficacy and efficacy• Cost and feasibility of routine laboratory monitoring, including WBC differential and reticulocytes
• Low cost compared with alternatives• Inability to measure quantitative %HbF
Stem cell transplantation• Potential cure• Lack of facilities and clinical expertise
• Availability of full siblings, which increases the chance of HLA matching
• Limited technology for HLA typing, cell processing, and preparation
• Inadequate supportive care (antibiotics, transfusions, isolation rooms)
• High risk of morbidity (graft versus host disease) and mortality
• Extremely high cost
  • HLA, human leukocyte antigen; WBC, white blood cell.