• M E Houwing, P J de Pagter, E J van Beers, B J Biemond, E Rettenbacher, A W Rijneveld, E M Schols, J N J Philipsen, R Y J Tamminga, K Fijn van Draat, E Nur, M H Cnossen, and SCORE Consortium.
• Department of Paediatric Haematology, Erasmus University Medical Center - Sophia Children's Hospital, Wytemaweg 80, 3015, CN, Rotterdam, the Netherlands. Electronic address: M.houwing@erasmusmc.nl.
• Blood Rev. 2019 Sep 1; 37: 100580.

AbstractSickle cell disease is an autosomal recessive, multisystem disorder, characterised by chronic haemolytic anaemia, painful episodes of vaso-occlusion, progressive organ failure and a reduced life expectancy. Sickle cell disease is the most common monogenetic disease, with millions affected worldwide. In well-resourced countries, comprehensive care programs have increased life expectancy of sickle cell disease patients, with almost all infants surviving into adulthood. Therapeutic options for sickle cell disease patients are however, still scarce. Predictors of sickle cell disease severity and a better understanding of pathophysiology and (epi)genetic modifiers are warranted and could lead to more precise management and treatment. This review provides an extensive summary of the pathophysiology and management of sickle cell disease and encompasses the characteristics, complications and current and future treatment options of the disease.Copyright © 2019. Published by Elsevier Ltd.

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