Cost effective nutritional therapy for managing sickle cell disease complications

Sickle cell diseaseSCDis a group of widespread single gene disorders and represents a significant public health burden worldwideIn the United Statesmost individuals with SCD are of African or Hispanic ancestrygroups often affected by healthcare disparitiesIncreased hemolysis of the fragilesickleshaped erythrocytesred blood cellsis characteristic of SCDThese red cells are destroyed and continuously removed from the circulationAssociated with a resultant increase of red blood cell production are two energy consuming eventsfaster protein synthesis andincreased protein catabolismThe increased protein metabolism results in abnormally high resting energy expenditureREEThis evidence suggests that patients with SCD require more dietary protein and energy than individuals without the diseaseHoweverto date the use of nutritional supplementation for addressing these complicationsis not adequately researchedand there are no recommended dietary allowances for SCDWe have successfully conducted a pilot randomized placebocontrolled supplementation trial in children with sickle cell anemiaSCAwho inherit two S allelesagedtoyearsWe used a nutritional formula designed to replace established nutrient shortagesincluding calorie consumption and essential amino acids with anti inflammatory effectsThe results show that plasma amino acid levels increasede gL Arginine bylean body mass improved bybone mineral content byand bone mineral density byREE was reduced byBased on these findingswe hypothesize that dietary protein and energy supplements will provide the extra nutrients known to be deficient in individuals with SCAThese supplements will promote tissue repair and lean body mass accretionand improve the clinical condition of individuals with SCAThe aims of this proposal are toaconduct a larger clinical trial to validate the effect of the nutritional supplement and its use for normalizing body massREEprotein metabolism and inflammationover six monthsin children with SCD andbConduct a robust compliance assessment intervention as a vital part of the studyNew innovative therapies are needed to treat SCDA cost effective self administered non toxic nutritional treatment should be made availableThis dietary approach could complement the standard patient care and therefore address both nutritional and hematological issues in tandemNutritional interventionfood supplementationis virtually non toxic andearly implementation could prevent characteristic under nutrition in individuals with SCDwho can be at risk even prenatally Sickle cell anemiaSCAaffects more thanpeople in the United States and represents a significant public health burden worldwideSickle cell patients have nutritional complications such as growth delayshort height and extremely reduced muscle masswhich are not being adequately treatedThis application proposes to use a targeted dietary supplementwhichif successfulwill reduce the nutritional issues that contribute to frequent ill health and substantial hospitalization costs in this patient population