title: The safety and efficacy of hydroxycarbamide in infants with sickle cell anemia.
abstract: 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.
authors.affiliation: Department of Paediatric Haematology, King's College Hospital NHS Foundation Trust, Denmark Hill, London, SE5 9RS, UK.
authors.forename: Clare
authors.initials: C
authors.lastname: Wykes
authors.forename: David C
authors.initials: DC
authors.lastname: Rees