Table 2.

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

Treatment	Advantages and indications	Disadvantages 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.