The effect of the
testosterone derivative
oxymetholone alone or in combination with the H1-receptor antagonist
ketotifen, which has recently been shown to block tumour
necrosis factor alpha (
TNF alpha), on
weight gain and performance status in human immunodeficiency virus (HIV) patients with chronic
cachexia was evaluated in a 30-week prospective pilot study. Thirty patients were randomly assigned to either
oxymetholone monotherapy (n 14) or
oxymetholone plus
ketotifen (n 16). Patients receiving treatment were compared with a group of thirty untreated matched controls, who met the same inclusion criteria.
Body weight and the Karnofsky index, which assesses the ability to perform activities of daily life, and several quality-of-life variables were measured to evaluate response to
therapy. The average
weight gain at peak was 8.2 (SD 6.2) kg (+ 14.5% of
body weight at study entry) in the
oxymetholone group (P < 0.001), and 6.1 (SD 4.6) kg (+10.9%) in the combination group (P < 0.005), compared with an average
weight loss of 1.8 (SD 0.7) kg in the untreated controls. The mean time to peak weight was 19.6 weeks in the monotherapy group and 20.8 weeks in the combination group. The Karnofsky index improved equally in both groups from 56% before to 67% after 20 weeks of treatment (P < 0.05). The quality of life variables (activities of daily life, and appetite/nutrition) improved in 68% (P < 0.05) and 91% (P < 0.01) of the treated patients respectively.
Oxymetholone was safe and promoted
weight gain in cachectic patients with advanced HIV-1
infection. The addition of
ketotifen did not further support
weight gain. These results suggest the need for a randomized, double-blind, placebo-controlled multicentre trial.