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Treatment of sickle cell disease: Beyond hydroxyurea

India is home to a large number of patients with sickle cell disorders. We do not have any clear data on its prevalence and incidence but can surmise that it remains an area of concern from several single-center data and regional papers. It roughly correlates in distribution with tribal populations and affects a large population with basic medical access, poor infant mortality rates, and life expectancy. Therefore, it has remained unrecognized as a public health problem in this country. Hydroxyurea remains the cornerstone of sickle cell management by pharmacological means and does change the natural history of the disease. There remain barriers to the widespread use of this drug, most commonly due to a fear of teratogenicity. It does not reduce the risks and effects of long-term organ damage. An understanding of the mechanisms behind the phenotypic presentation of the disease has opened research into several medications targeting different mechanisms. This review looks at the newer drugs that have been approved for sickle cell disease in addition to reviewing the data on hydroxyurea.