Randomized, Double-blind, Placebo Controlled Phase III Trial of Oxymetholone for the Treatment of HIV Wasting and Lipodystrophy

Purpose of the Study

Although HAART has greatly impacted treatment of HIV infection, lipodystrophy and HIV wasting still represent unsolved problems in HIV therapy and patient care. Oxymetholone, a testosterone derivative, has been shown to promote weight gain in AIDS-associated wasting.

Methods

We analyzed the effects of oxymetholone [50 mg BII) and TII)] in a randomized (1:1:1), double-blind, placebo-controlled phase III study with 92 subjects (all on ART) experiencing unintended weight loss>10% of ideal weight according to Broca with special emphasis on body composition measurement 80 patients (69 men,11 women, mean age:38,8 years) completed the 16-week double-blind study phase.

Results

Mean weight gain was +3.7 ± 3.5 kg and +3.1 ± 2.7 kg in the oxymetholone groups [BII); n = 25 vs TII); n = 27] as opposed to +0.97 ± 3.4 kg in the placebo were observed in body cell mass (30.6 before vs 32.5 after therapy), lean body mass [56.3 before vs 59.0 after therapy in the BII) group] and body mass index (21.4 before vs 22.1 after therapy) exclusively in oxymetholone-treated patients. The extracellular mass to body cell mass ratio,(p < 0,0001). Total body fat was unchanged by oxymetholone treatment. Adverse events were mainly hepatic occurring in 14% of oxymetholone-treated patients with significant elevations of AST, ALT and GGT;2 patients (7.4%) in the BII) arm experienced grade 3 and 4 liver toxicity compared with 6 [21.4%) in the TII] arm.

Conclusion

Oxymetholone was found to have true anabolic effects in a double-blind, placebo-controlled phase III trial. The (BII) (100 mg/d) regimen appeared equally effective to TII) (150 mg/d) dosing while displaying reduce liver toxicity. Due to its favourable protein anabolism, it may be recommended for therapy of wasting and lipodystrophy I HIV-infected subjects.