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 |
| 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.