Efficacy, effectiveness, and toxicities of hydroxyurea for adults with sickle cell disease (SCD), primarily based on data from the Multicenter Study of Hydroxyurea (MSH) randomized clinical trial. Adapted from Lanzkron et al16 and Brawley et al.18
| Criterion . | Outcome . | Evidence grade . |
|---|---|---|
| Efficacy/Effectiveness | ||
| %HbF | Increased | High |
| Pain episodes | Decreased | High |
| Hospitalization | Decreased | High |
| Transfusions | Decreased | High |
| Mortality | Decreased | Low |
| Neurological events | Decreased | Insufficient |
| Toxicity | ||
| Leg ulcers | Comparable | High |
| Leukemia | Comparable | Low |
| Spermatogenesis | Defects | Low |
| Skin neoplasms | Comparable | Insufficient |
| Secondary cancer | Comparable | Insufficient |
| Adverse pregnancy | Comparable | Insufficient |
| Criterion . | Outcome . | Evidence grade . |
|---|---|---|
| Efficacy/Effectiveness | ||
| %HbF | Increased | High |
| Pain episodes | Decreased | High |
| Hospitalization | Decreased | High |
| Transfusions | Decreased | High |
| Mortality | Decreased | Low |
| Neurological events | Decreased | Insufficient |
| Toxicity | ||
| Leg ulcers | Comparable | High |
| Leukemia | Comparable | Low |
| Spermatogenesis | Defects | Low |
| Skin neoplasms | Comparable | Insufficient |
| Secondary cancer | Comparable | Insufficient |
| Adverse pregnancy | Comparable | Insufficient |