Key considerations when discussing HU with an adult with SCD
| HU should be discussed with all adults with SCD. |
| HU reduced acute SCD complications in a placebo-controlled randomized trial.54 Adults with SCA with ≥3 severe pain crises in a year, with pain that interferes with daily activities, or with severe or recurrent ACS should be treated with HU. |
| Long-term use of HU in nonrandomized studies demonstrated enhanced survival in patients with HbSS/HbSβ0 thalassemia disease.55,56 |
| HU improves Hb and reduces the frequency of blood transfusion and should be offered to patients with symptomatic anemia. |
| There is insufficient evidence about the efficacy of HU in primary or secondary stroke prevention in adults. |
| HU should be offered to patients with SCN and should be given in combination with ESAs if there is renal-related anemia. |
| There is a lack of evidence from randomized controlled trials about the beneficial effect of HU on progression of end organ damage in adults. The increase in Hb with an accompanying increase in HbF could only be beneficial; thus, HU can be considered in PH, priapism, and chronic hypoxia, but the benefits and risks should be discussed thoroughly with patients. |
| The majority of evidence pertains to patients with HbSS/Sβ0 thalassemia, so most recommendations are for these genotypes. However, adults with HbSC should be offered HU if they have frequent acute pain or ACS. |
| HU may have a detrimental effect on spermatogenesis and may be teratogenic. Male patients should be considered for sperm analysis and cryopreservation prior to starting HU treatment. Male and female patients are advised to use contraception while on HU and to stop 3 months preconception. In women whose disease severity is alleviated by HU and are not able to receive blood transfusion, clinicians may consider ongoing HU therapy during pregnancy, with provision of appropriate counseling about fetal risks. |
| HU has also recently been found to be relatively safe in settings with high infectious disease burden, increasing our confidence in broadening the clinical use of this drug.57,58 |
| HU should be discussed with all adults with SCD. |
| HU reduced acute SCD complications in a placebo-controlled randomized trial.54 Adults with SCA with ≥3 severe pain crises in a year, with pain that interferes with daily activities, or with severe or recurrent ACS should be treated with HU. |
| Long-term use of HU in nonrandomized studies demonstrated enhanced survival in patients with HbSS/HbSβ0 thalassemia disease.55,56 |
| HU improves Hb and reduces the frequency of blood transfusion and should be offered to patients with symptomatic anemia. |
| There is insufficient evidence about the efficacy of HU in primary or secondary stroke prevention in adults. |
| HU should be offered to patients with SCN and should be given in combination with ESAs if there is renal-related anemia. |
| There is a lack of evidence from randomized controlled trials about the beneficial effect of HU on progression of end organ damage in adults. The increase in Hb with an accompanying increase in HbF could only be beneficial; thus, HU can be considered in PH, priapism, and chronic hypoxia, but the benefits and risks should be discussed thoroughly with patients. |
| The majority of evidence pertains to patients with HbSS/Sβ0 thalassemia, so most recommendations are for these genotypes. However, adults with HbSC should be offered HU if they have frequent acute pain or ACS. |
| HU may have a detrimental effect on spermatogenesis and may be teratogenic. Male patients should be considered for sperm analysis and cryopreservation prior to starting HU treatment. Male and female patients are advised to use contraception while on HU and to stop 3 months preconception. In women whose disease severity is alleviated by HU and are not able to receive blood transfusion, clinicians may consider ongoing HU therapy during pregnancy, with provision of appropriate counseling about fetal risks. |
| HU has also recently been found to be relatively safe in settings with high infectious disease burden, increasing our confidence in broadening the clinical use of this drug.57,58 |
Based on National Heart, Lung, and Blood Institute guidelines.29