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Chronic organ injuries in children with sickle cell disease.

Authors:
Slimane Allali Melissa Taylor Joséphine Brice Mariane de Montalembert

Haematologica 2021 Feb 25. Epub 2021 Feb 25.

Department of General Pediatrics and Pediatric Infectious Diseases, Reference Center for Sickle Cell Disease, Necker Hospital for Sick Children, Assistance Publique - Hôpitaux de Paris (AP-HP), Université de Paris, Paris; Laboratory of Excellence GR-Ex, Paris; Institut National de la Transfusion Sanguine (INTS), Université de Paris, Inserm U1134, Paris.

Median life expectancy of patients with sickle cell disease has increased to up to 55 years but there are still frequent cases of premature death, mostly in patients with pre-existing organ failure such as pulmonary hypertension, kidney injury, and cerebral vasculopathy. Most organ injuries remain asymptomatic for a long time and can only be detected through early systematic screening. Protocols combining assessment of velocities on transcranial Doppler and regular transfusions in patients with abnormal velocities have been demonstrated to dramatically reduce the risk of stroke. In contrast, no consensus has been reached on systematic screening or therapy for silent cerebral infarcts. The prognostic significance of increased tricuspid regurgitant jet velocity on echocardiography has not yet been identified in children, whereas increased albuminuria is a good predictor of kidney injury. Finally, screening for hip and eye disorder is recommended; however, different countries adopt different screening strategies. Hydroxyurea is probably of potential benefit in preventing chronic organ damage but this requires further study in order to be fully demonstrated. Efficacy and safety of the other new drugs available are also under investigation.

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http://dx.doi.org/10.3324/haematol.2020.271353DOI Listing
February 2021

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