Table 3

Cumulative frequency of adverse laboratory events among children and adolescents with SCA treated at MTD in the phase 1/2 HUG-KIDS study21 

ToxicityToxicity definitionPercentage of patientsPercentage of visits
Neutropenia 2.0 × 109/L 67 5.2 
Reticulocytopenia 80 × 109/L unless hemoglobin ≥ 9.0 g/dL 42 1.6 
Anemia 20% decrease or < 4.5 g/dL 32 1.1 
ALT elevation 2× upper limit of normal 13 0.4 
Thrombocytopenia 80 × 109/L 0.3 
Creatinine elevation 50% increase and > 1.0 mg/dL 0.0 
ToxicityToxicity definitionPercentage of patientsPercentage of visits
Neutropenia 2.0 × 109/L 67 5.2 
Reticulocytopenia 80 × 109/L unless hemoglobin ≥ 9.0 g/dL 42 1.6 
Anemia 20% decrease or < 4.5 g/dL 32 1.1 
ALT elevation 2× upper limit of normal 13 0.4 
Thrombocytopenia 80 × 109/L 0.3 
Creatinine elevation 50% increase and > 1.0 mg/dL 0.0 

SCA indicates sickle cell anemia; MTD, maximum tolerated dose; HUG-KIDS, prospective National Heart, Lung and Blood Institute–sponsored pediatric trial; and ALT, alanine aminotransferase.

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