Characteristics and considerations when selecting DMMs for youths with SCD
| Considerations . | Disease-modifying medications . | |||||
|---|---|---|---|---|---|---|
| . | Hydroxyurea . | L-glutamine . | Voxelotor* . | Crizanlizumab . | ||
| Patient characteristics | Age | <4 years | + | − | − | − |
| ≥4 years | + | +^ | + | − | ||
| ≥16 years | + | + | + | + | ||
| Genotype | HbSS/Sβ0 | + | + | + | + | |
| HbSC | −† | − | +‡ | + | ||
| HbSβ+ | − | + | +‡ | + | ||
| Patient preference | Administration | Intravenous | − | − | − | + |
| Oral | + | + | + | − | ||
| Potential side effects | Cytopenias | + | − | − | − | |
| Hair thinning | + | − | − | − | ||
| Gastrointestinal | + | + | + | + | ||
| Allergic reaction/rash | − | − | + | + | ||
| Pain | − | + | − | + | ||
| Impaired fertility | + | − | − | − | ||
| Monitoring | Laboratory monitoring | + | − | − | − | |
| Availability | Available at pharmacy | +§ | − | − | N/A | |
| Intended outcome | Clinical | Reduce VOC | + | + | − | + |
| Reduce ACS | + | + | − | + | ||
| Reduce stroke risk | + | − | − | − | ||
| Improve quality of life | + | − | − | − | ||
| Reduce mortality | + | − | − | − | ||
| Hematologic | Improve anemia | + | − | + | − | |
| Other | Access | Cost | − | + | + | + |
| Evidence | >1 positive clinical trial | + | − | + | − | |
| Considerations . | Disease-modifying medications . | |||||
|---|---|---|---|---|---|---|
| . | Hydroxyurea . | L-glutamine . | Voxelotor* . | Crizanlizumab . | ||
| Patient characteristics | Age | <4 years | + | − | − | − |
| ≥4 years | + | +^ | + | − | ||
| ≥16 years | + | + | + | + | ||
| Genotype | HbSS/Sβ0 | + | + | + | + | |
| HbSC | −† | − | +‡ | + | ||
| HbSβ+ | − | + | +‡ | + | ||
| Patient preference | Administration | Intravenous | − | − | − | + |
| Oral | + | + | + | − | ||
| Potential side effects | Cytopenias | + | − | − | − | |
| Hair thinning | + | − | − | − | ||
| Gastrointestinal | + | + | + | + | ||
| Allergic reaction/rash | − | − | + | + | ||
| Pain | − | + | − | + | ||
| Impaired fertility | + | − | − | − | ||
| Monitoring | Laboratory monitoring | + | − | − | − | |
| Availability | Available at pharmacy | +§ | − | − | N/A | |
| Intended outcome | Clinical | Reduce VOC | + | + | − | + |
| Reduce ACS | + | + | − | + | ||
| Reduce stroke risk | + | − | − | − | ||
| Improve quality of life | + | − | − | − | ||
| Reduce mortality | + | − | − | − | ||
| Hematologic | Improve anemia | + | − | + | − | |
| Other | Access | Cost | − | + | + | + |
| Evidence | >1 positive clinical trial | + | − | + | − | |
Patient characteristics and intended outcome considerations are from published clinical trial data.
Voxelotor has been withdrawn from the market because of safety concerns and is not currently available to treat SCD.
L-glutamine is for ages ≥5 years.
The trial of hydroxyurea in youths with HbSC disease was not completed.
Voxelotor has not been studied in children <12 years old with HbSC and HbSβ+.
Liquid hydroxyurea may be more challenging to access, since it must be compounded.
N/A, not applicable.