Oral Hydroxycarbamide Alleviates Pain in Infants with Sickle Cell Anemia

Early treatment reduces complications of sickle cell disease with little toxicity.

Complications of sickle cell anemia can begin early in life. Hydroxycarbamide (hydroxyurea) reduces complications in adults with sickle cell disease, but its efficacy in young children has not been determined. In a multicenter, double-blind trial, researchers randomized 193 infants (age range, 9–18 months) with sickle cell anemia to receive oral liquid hydroxycarbamide (20 mg/kg/day) or placebo for 2 years.

Although no significant differences were observed between groups in spleen function or mean glomerular filtration rate (the primary endpoints), hydroxycarbamide was associated with significantly fewer episodes of pain (defined as duration >2 hours and requiring analgesia) and dactylitis. Treatment with hydroxycarbamide also showed evidenc…

Reviewing Author

F. Bruder Stapleton, MD

Disclosures

Consultant/Advisory BoardCareer Physician

Editorial BoardsUpToDate

Leadership Positions in Professional SocietiesAmerican Society of Pediatric Nephrology Foundation (Chair)

F. Bruder Stapleton, MD

Disclosures

Consultant/Advisory BoardCareer Physician

Editorial BoardsUpToDate

Leadership Positions in Professional SocietiesAmerican Society of Pediatric Nephrology Foundation (Chair)