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Randomised controlled trial
Hydroxycarbamide for very young children with sickle cell anaemia: no effect on the primary outcomes of spleen or kidney function, but evidence for decreased pain and dactylitis, with minimal toxicity
  1. Deepa Manwani
  1. Department of Pediatrics, Albert Einstein College of Medicine, Children's Hospital at Montefiore, Bronx, New York, USA
  1. Correspondence to Deepa Manwani
    Department of Pediatrics, Albert Einstein College of Medicine, Children's Hospital at Montefiore, 3415 Bainbridge Avenue, Bronx, NY 10467, USA; dmanwani{at}montefiore.org

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Context

Sickle cell anaemia (SCA) is a disorder of haemoglobin polymerisation that results in vaso-occlusion and haemolytic anaemia, culminating in organ injury and early mortality. Elevated fetal haemoglobin has been associated with a less severe phenotype leading to an interest in hydroxycarbamide (also known as hydroxyurea) use. The MSH study1 demonstrated that hydroxycarbamide in adults with severe sickle cell disease reduced painful events, hospitalisations, acute chest syndrome and the number of blood transfusions. The HUG–KIDS study,2 a phase I/II study, demonstrated safety and haematologic …

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  • Competing interests None.