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Blood Rev. 2019 May 20. pii: S0268-960X(18)30115-2. doi: 10.1016/j.blre.2019.05.004. [Epub ahead of print]

Sickle cell disease: Clinical presentation and management of a global health challenge.

Author information

1
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.
2
Department of Paediatric Haematology, Erasmus University Medical Center - Sophia Children's Hospital, Wytemaweg 80, 3015, CN, Rotterdam, the Netherlands. Electronic address: P.depagter@erasmusmc.nl.
3
Department of Internal Medicine and Dermatology, Van Creveldkliniek, University Medical Center Utrecht, Internal mail no C.01.412, 3508, GA, Utrecht, the Netherlands. Electronic address: E.j.vanbeers-3@umcutrecht.nl.
4
Department of Internal Medicine and Clinical Haematology, Amsterdam University Medical Centers, Meibergdreef 9, 1105, AZ, Amsterdam, the Netherlands. Electronic address: B.j.biemond@amc.uva.nl.
5
Department of Paediatric Haematology, Radboud University Medical Center - Amalia Children's Hospital, Geert Grooteplein Zuid 10, 6500, HB, Nijmegen, the Netherlands. Electronic address: Eva.rettenbacher@radboudumc.nl.
6
Department of Haematology, Erasmus University Medical Center, Wytemaweg 80, 3015, CN, Rotterdam, the Netherlands. Electronic address: A.rijneveld@erasmusmc.nl.
7
Department of Haematology, Radboud University Medical Center, Geert Grooteplein Zuid 10, 6525, GA, Nijmegen, the Netherlands. Electronic address: Saskia.schols@radboudmumc.nl.
8
Department of Cell Biology, Erasmus University Medical Center, Wytemaweg 80, 3015, CN, Rotterdam, the Netherlands. Electronic address: J.philipsen@erasmusmc.nl.
9
Department of Paediatric Oncology and Haematology, University Medical Center Groningen - Beatrix Children's Hospital, Postbus 30001, 9700, RB, Groningen, the Netherlands.. Electronic address: R.y.j.tamminga@umcg.nl.
10
Department of Paediatric Haematology, Amsterdam University Medical Centers - Emma Children's Hospital, Meibergdreef 9, 1105 AZ Amsterdam, the Netherlands; Department of Plasma Proteins, Sanquin Research, the Netherlands. Electronic address: C.j.fijnvandraat@amc.uva.nl.
11
Department of Internal Medicine and Clinical Haematology, Amsterdam University Medical Centers, Meibergdreef 9, 1105, AZ, Amsterdam, the Netherlands. Electronic address: E.nur@amc.uva.nl.
12
Department of Paediatric Haematology, Erasmus University Medical Center - Sophia Children's Hospital, Wytemaweg 80, 3015, CN, Rotterdam, the Netherlands. Electronic address: M.cnossen@erasmusmc.nl.

Abstract

Sickle 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.

KEYWORDS:

Complications; Sickle cell disease; Treatment; Vaso-occlusion

PMID:
31128863
DOI:
10.1016/j.blre.2019.05.004

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