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Hydroxyurea Therapy in UK Children with Sickle Cell Anaemia – A Single Centre Experience

Phillips, KLE, Healy, L, Smith, L and Keenan, RD Hydroxyurea Therapy in UK Children with Sickle Cell Anaemia – A Single Centre Experience. Pediatric Blood and Cancer. ISSN 1545-5009 (Accepted)

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Despite the demonstrated efficacy of hydroxyurea therapy, children with sickle cell anaemia in the United Kingdom (UK) are preferentially managed with supportive care or transfusion. Hydroxyurea is reserved for children with severe disease phenotype. This is in contrast to North America and other countries where hydroxyurea is widely used for children of all clinical phenotypes. The conservative UK practice may in part be due to concerns about toxicity, in particular marrow suppression with high doses, and growth in children. We monitored 37 paediatric patients with sickle cell anaemia who were treated with hydroxyurea at a single UK treatment centre. Therapy was well tolerated and mild transient cytopenias were the only toxicity observed. Comparative analysis of patients receiving ≥26mg/kg/day versus <26 mg/kg/day demonstrates increasing dose has a significant positive effect on foetal haemoglobin (29.2% v 20.4%, p=0.0151), mean cell volume (94.4 v 86.5, p=0.0183) and reticulocyte count (99.66 x109/L v 164.3x109/L, p=0.0059). Marrow suppression was not a clinical problem with high dose treatment, haemoglobin 92.25 g/L v 91.81 g/L (ns), neutrophil count 3.3 x109/L v 4.8 x109/L (ns) and platelet count 232.4 x109/L v 302.2 x109/L (ns). Normal growth rates were maintained in all children. Good adherence to therapy was a significant factor in reducing hospitalisations This study demonstrates the effectiveness and safety in practice of high dose hydroxyurea as a disease modifying therapy which we advocate for all children with sickle cell anaemia.

Item Type: Article
Uncontrolled Keywords: 1112 Oncology And Carcinogenesis, 1114 Paediatrics And Reproductive Medicine
Subjects: R Medicine > RM Therapeutics. Pharmacology
Divisions: Pharmacy & Biomolecular Sciences
Publisher: Wiley
Date Deposited: 15 Sep 2017 10:17
Last Modified: 15 Sep 2017 10:17
DOI or Identification number: 10.1002/pbc.26833
URI: http://researchonline.ljmu.ac.uk/id/eprint/7101

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