JAMA : the journal of the American Medical Association
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Review Practice Guideline
Management of sickle cell disease: summary of the 2014 evidence-based report by expert panel members.
Sickle cell disease (SCD) is a life-threatening genetic disorder affecting nearly 100,000 individuals in the United States and is associated with many acute and chronic complications requiring immediate medical attention. Two disease-modifying therapies, hydroxyurea and long-term blood transfusions, are available but underused. ⋯ Hydroxyurea and transfusion therapy are strongly recommended for many individuals with SCD. Many other recommendations are based on quality of evidence that is less than high due to the paucity of clinical trials regarding screening, management, and monitoring for individuals with SCD.
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Randomized Controlled Trial
Ten-year follow-up after initiation of statin therapy in children with familial hypercholesterolemia.