| Potential benefit | Comments |
| Improved survival and increased life expectancy | - Especially useful in reducing death from pulmonary complications including ACS and pulmonary hypertension
- Longer duration of therapy associated with cumulative benefit
|
| Decreased vaso-occlusive pain | - Applies to infants, children, and adults
- Can reduce the number of episodes by 50% or more
- In turn reduces hospitalizations
|
| Decreased ACS episodes | - Applies to infants, children, and adults
- Can reduce the number of episodes by 50% or more
- In turn reduces hospitalizations
|
| Decreased transfusions | - In turn reduces transfusion complications including alloimmunization, transfusion reactions, and iron overload
|
| Decreased TCD measurements | - Correlates with reduced risk of stroke
- Generally applies to children and adolescents
- Regular transfusions are standard of care for most children with high TCD
|
| Decreased priapism episodes | - Indirect evidence, mostly from small observational studies
|
| Improved quality of life and daily functioning | - Involves multiple benefits such as those listed above
- May translate to fewer hospitalizations, better school attendance, and fewer days lost from work
|
| Improved disease markers | - Increased cerebral oxygen saturation
- Increased fetal hemoglobin (decreased percent HbS)
- Increased total hemoglobin (decreased severity of anemia)
- Improved hemoglobin-oxygen saturation
- Improved markers kidney function
- Improved markers of cardiac and pulmonary disease
- Reduced hemolysis
|
