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Br J Haematol. 2013 Aug;162(4):455-64. doi: 10.1111/bjh.12413. Epub 2013 Jun 15.

Sickle cell disease: time for a closer look at treatment options?

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Division of Pediatric Hematology and Oncology, Weill Cornell Medical College, New York, NY 10065, USA.


Tremendous progress has been made in the care of individuals with sickle cell over the past several decades. Major successes have been comprehensive infection prophylaxis, prediction and prevention of stroke, and better transfusion care, the latter including both prevention of alloimmunization and treatment of iron overload. However, definitive therapies remain limited to hydroxycarbamide (hydroxyurea) and stem cell transplantation, both of which have been in use for at least two decades. Despite knowing the progressive natural history of the disease with organ dysfunction, failure, and ultimately death at a young age, definitive therapies are considered for only a small proportion of individuals. Consequently, while life expectancy has improved dramatically from the last century, the ongoing pace of advancement has slowed or stalled. We believe that it is time to broaden the use of definitive therapy for those with asymptomatic disease, being cautiously more aggressive in our approach.


hydroxycarbamide; morbidity; sickle cell disease; stem cell transplantation

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