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Current Pharmaceutical Design


ISSN (Print): 1381-6128
ISSN (Online): 1873-4286

Pharmacotherapy of Sickle Cell Disease in Children

Author(s): Kathleen A. Neville and Julie A. Panepinto

Volume 21, Issue 39, 2015

Page: [5660 - 5667] Pages: 8

DOI: 10.2174/1381612821666151030105213

Price: $65


Sickle cell disease (SCD) is a potentially devastating and life threatening condition that is caused by an autosomal recessive inherited hemoglobinopathy which results in vaso-occlusive phenomena and hemolysis. The severity of this disorder is widely variable, but overall mortality is increased and life expectancy decreased when compared to the general population.

Care of patients with sickle cell disease is largely supportive. In fact, hydroxyurea is the only drug used that modifies disease pathogenesis. Painful vaso-occlusive events are the most common complication experienced by both children and adults with sickle cell disease and hydroxyurea is the only treatment option available to prevent the development of these events. Most events are managed with traditional supportive care measures (i.e. aggressive hydration, antiinflammatory and narcotic analgesics) that have not changed in decades. As such, there is an overwhelming need for both the development of new agents and new approaches to treatment with existing modalities for patients with sickle cell disease.

Keywords: Sickle cell disease, therapeutics, treatment, children.

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