Evaluation of the effects of oxandrolone on malnourished HIV-positive pediatric patients

Objective. To determine the safety and efficacy of anabolic therapy to prev ent or reverse wasting and malnutrition in human immunodeficiency virus (HI V)-infected pediatric patients. The anabolic steroid, oxandrolone, was eval uated because of its safe and effective use in other pediatric conditions. Methods. Nine HIV-positive children who were malnourished or at risk for ma lnutrition (4 females, 5 males; 4-14 years of age) took oxandrolone for 3 m onths (.1 mg/kg/day orally). Quantitative HIV ribonucleic acid polymerase c hain reaction and CD4(+) T-cell levels, complete blood cell count (CBC) and chemistry profile, endocrinologic studies, resting energy expenditure, res piratory quotient, nutritional measures, body composition assessment with q uantitative computed tomography, and skinfold body composition measurements were determined before treatment, during treatment (3 months), and for 3 m onths after treatment. Statistical analyses were completed using the Friedm an two-way analysis of variance and Spearman correlation tests. Results. No adverse clinical or laboratory events or changes in Tanner stag ing or virilization occurred. Quantitative HIV ribonucleic acid polymerase chain reaction and CD4(+) T-cell levels did not change significantly. Insul in-like growth factor 1 increased, suggesting an anabolic effect of treatme nt. The rate of weight gain increased during treatment and was maintained a fter treatment. Linear growth continued and was maintained throughout treat ment, whereas bone age did not increase significantly. Anthropometric asses sments indicated an increase in muscle mass and a decrease in fat while pat ients were on treatment, and a mild decrease of muscle and increased fat po sttreatment. Likewise, computed tomography scan results demonstrated simila r changes in muscle mass. Resting energy expenditure and respiratory quotie nt remained stable throughout treatment and follow-up. No significant chang es were seen in the quality of life questionnaire. Conclusions. Treatment with oxandrolone for 3 months in HIV-infected childr en was well-tolerated, safe, and associated with markers of anabolism. The latter effect was maintained partially for 3 months after discontinuation o f a 3-month course of therapy. Additional studies are needed to assess the potential benefits and risks of a longer course of therapy or a higher dose of oxandrolone in HIV-infected children.