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2011•Expert Review of HematologyRequires access

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

Clare Wykes, David C. Rees

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Abstract

Evaluation of: Wang WC, Ware RE, Miller ST et al. Hydroxycarbamide in very young children with sickle-cell anaemia: a multicentre, randomised, controlled trial (BABY HUG). Lancet 377(9778), 1663–1672 (2011).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.

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What this paper is about

Evaluation of: Wang WC, Ware RE, Miller ST et al. Hydroxycarbamide in very young children with sickle-cell anaemia: a multicentre, randomised, controlled trial (BABY HUG). Lancet 377(9778), 1663–1672 (2011).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.

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Available abstract

Evaluation of: Wang WC, Ware RE, Miller ST et al. Hydroxycarbamide in very young children with sickle-cell anaemia: a multicentre, randomised, controlled trial (BABY HUG). Lancet 377(9778), 1663–1672 (2011).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.

Key concepts: Medicine, Hydroxycarbamide, Sickle cell anemia, Anemia, Hemoglobinopathy, Intensive care medicine, Pediatrics, Hemolytic anemia

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