Table 3.

Hematological responses to hydroxyurea treatment in patients with sickle cell anemia. Studies in which hydroxyurea was escalated to maximum tolerated dose (MTD) usually averaged > 25 mg/kg/day and achieved laboratory thresholds of Hb > 9 g/dL, MCV > 100 fL, and HbF ~20%. In contrast, studies in which hydroxyurea was not escalated to MTD usually averaged 20 mg/kg/day and achieved Hb < 9 g/dL, MCV < 100 fL, and HbF ~15%.

Year# of patientsAverage age, yMaximum tolerated doseAvg dose, mg/kg/dTreatment duration, yHb, g/dLMCV, fLHbF, %WBC, x 109/LANC, x 109/LRef
Unpub indicates unpublished St. Jude results; NA, not available 
1992 32 27.6 Yes 21.3 0.8 9.7 117 15 8.4 4.6 5  
1999 71 9.8 Yes 25.6 1.5 9.1 102 16.3 9.1 4.4 7  
2004 106 10.3 Yes 25.9 3.8 9.5 107 19.7 7.2 3.6 10  
2005 11 3.4 Yes 30.0 9.0 96 23.3 8.9 NA 28  
2007 37 6.8 Yes 27.9 0.8 9.4 104 22.7 NA NA 31  
2009 14 3.9 Yes 28 2.1 9.5 99 25.9 NA 3.0 26  
2009 111 7.6 Yes 26.7 3.2 9.7 107 23.2 7.5 3.8 Unpub 
1996 22 No ~20 0.5 8.5 96 15.3 8.9 NA 19  
2001 21 1.3 No ~20 2.0 8.8 90 20.3 NA 4.2 8  
2001 22 No ~20 8.7 97 12.9 NA 4.0 9  
2005 32 No ~20 8.8 92 12.5 NA 4.6 32  
Year# of patientsAverage age, yMaximum tolerated doseAvg dose, mg/kg/dTreatment duration, yHb, g/dLMCV, fLHbF, %WBC, x 109/LANC, x 109/LRef
Unpub indicates unpublished St. Jude results; NA, not available 
1992 32 27.6 Yes 21.3 0.8 9.7 117 15 8.4 4.6 5  
1999 71 9.8 Yes 25.6 1.5 9.1 102 16.3 9.1 4.4 7  
2004 106 10.3 Yes 25.9 3.8 9.5 107 19.7 7.2 3.6 10  
2005 11 3.4 Yes 30.0 9.0 96 23.3 8.9 NA 28  
2007 37 6.8 Yes 27.9 0.8 9.4 104 22.7 NA NA 31  
2009 14 3.9 Yes 28 2.1 9.5 99 25.9 NA 3.0 26  
2009 111 7.6 Yes 26.7 3.2 9.7 107 23.2 7.5 3.8 Unpub 
1996 22 No ~20 0.5 8.5 96 15.3 8.9 NA 19  
2001 21 1.3 No ~20 2.0 8.8 90 20.3 NA 4.2 8  
2001 22 No ~20 8.7 97 12.9 NA 4.0 9  
2005 32 No ~20 8.8 92 12.5 NA 4.6 32  
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