Despite the availability of highly active antiretroviral therapy (HAART), chronic, involuntary weight loss still remains a serious problem for some people living with HIV. Various alterations in energy metabolism and endocrine regulation have been found to cause loss of lean body mass (LBM) and body cell mass (BCM).
Previously studies in HIV-positive men undergoing androgen replacement therapy or treatment with recombinant human growth hormone (rGH) have shown partial restoration of lean body mass, but these treatments have been largely ineffective in eugonadal individuals.
Now researchers from the Universities of Essen and Bonn have shown that the anabolic steroid oxymetholone can be considered an effective treatment for eugonadal male and female patients with AIDS-associated wasting.
A total of 89 patients (79 men and 10 women: 69 gay/bisexual men, 12 intravenous drug users, 7 heterosexual contact, one transfusion recipient) were enrolled
Patients were randomised to receive either 100mg/day twice daily (BID) or 150mg/day three times daily (TID) or placebo for 16 weeks. Patients in the placebo group gained 1.0 kg compared to 3.0kg among those receiving therapy three times daily and 3.5kg among those receiving therapy twice daily.
Seventeen patients (19%) discontinued treatment during the double-blind phase of the study. Fourteen patients (16%) were lost to follow-up or discontinued for personal or unknown reasons. One patient in the oxymetholone BID arm discontinued due to nausea and vomiting and two patients in the oxymetholone TID arm discontinued due to elevated liver enzymes.
Significant improvements were noted in appetite and food intake, increased well-being and reduced weakness by self-examination. Recent research has found that higher LBM is significantly associated with better physical functioning and better general health perceptions as well as with fewer days in bed in men, though not in women. The most important adverse event was liver associated toxicity. Overall, 35% of patients in the TID arm, 27% of patients in the BID arm and no patients in the placebo group had a greater than five times baseline increase for alanine aminotransferase during the double-blind phase of the study.
Weight gain was observed after an average of two weeks. The initial increase was rapid until week 4, but body weight remained at the same level from this point on. There was no correlation between the extent of weight gain and age, sex or disease stage.
Only one female developed a self-reported clitoris enlargement, whereas changes in libido were similar across groups.
Oxymetholone can be considered an effective anabolic steroid in eugonadal male and female patients with AIDS-associated wasting, with the twice daily 100mg/day regimen appearing equally effective as the three times daily 150mg/day in terms of weight gain, LBM and BCM, as well as being associated with less, but still significant liver toxicity.
The researchers recommended (as a guide for clinical practice) an induction period of 8 to 10 weeks with 50mg BID and thereafter ‘maintenance’ therapy with 50mg once daily or every other day since this regimen has shown sufficient weight and LBM gain while reducing hepatic side-effects.
Hengge U, Double-blind randomised, placebo-controlled phase III trial of oxymetholone for the treatment of HIV wasting. AIDS 2003; 17:699-710.