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scientific articles

The safety and efficacy of hydroxycarbamide in infants with sickle cell anemia

key information

source: Expert Review of Hematology

year: 2011

authors: Wykes C, Rees DC


The BABYHUG study is the first randomized controlled prospective trial of hydroxycarbamide in infants with sickle cell anemia to look at its effect on organ function. Almost 200 patients were recruited, irrespective of disease severity, over a 3-year period and followed for 2 years. The primary end points of splenic function and glomerular filtration rate were not reached. The trial did show a benefit in frequency of pain, dactylitis, acute chest syndrome, hospital admissions and transfusion requirements. Adverse effects were low.

organization: King's College Hospital NHS Foundation Trust, London

DOI: 10.1586/ehm.11.40

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