Low fixed-dose hydroxyurea in severely affected Indian children with sickle cell disease.
There is limited data on the efficacy of hydroxyurea (HU) in Indian sickle cell anemia patients who have severe manifestations despite high fetal hemoglobin (Hb F). Sixty sickle cell anemia children (5-18 years) with more than three episodes of vasoocclusive crises or blood transfusions per year were randomized to receive HU (n = 30) or placebo (n = 30) therapy. Fixed dose (10 mg/kg/day) of HU was administered for 18 months and the patients were followed-up monthly with clinical assessment and laboratory monitoring. In the HU group, hemoglobin (Hb) and Hb F levels increased significantly along with a significant decrease in the number of painful crises, blood transfusion requirements and hospitalizations compared to the placebo group. No major adverse events were observed in this study. In conclusion, low-fixed dose HU therapy was effective for the treatment of Indian sickle cell anemia children. However, there is a need for long-term studies to evaluate the efficacy and toxicity in a larger number of Indian sickle cell anemia patients.
Department of Paediatrics, Government Medical College, Nagpur, Maharsashtra-440009, India. dipty47@rediffmail.com
Dipty L
DL
Jain
Vijaya
V
Sarathi
Saumil
S
Desai
Manoj
M
Bhatnagar
Abhijit
A
Lodha
D000293:Adolescent; D000755:Anemia, Sickle Cell / Q000097:blood / Q000188:drug therapy*; D000986:Antisickling Agents / Q000009:adverse effects / Q000627:therapeutic use*; D001803:Blood Transfusion / Q000706:statistics & numerical data; D002648:Child; D005260:Female; D005319:Fetal Hemoglobin / Q000378:metabolism; D005500:Follow-Up Studies; D006760:Hospitalization / Q000706:statistics & numerical data; D006801:Humans; D006918:Hydroxyurea / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D010146:Pain / Q000517:prevention & control; D016896:Treatment Outcome
D000986:Antisickling Agents; D005319:Fetal Hemoglobin; D006918:Hydroxyurea