Thalidomide for the treatment of AIDS-associated wasting

A double-blind, placebo-controlled trial of efficacy and safety of thalidom ide in AIDS-associated wasting was carried out. Ninety-nine of 103 male pat ients had at least one on-study measurement (intent-to-treat [ITT] cohort). Patients were randomized to thalidomide at 100 mg/day (T-100) or 200 mg/da y (T-200), or placebo for 8 weeks. By ITT analysis, the mean change in body weight of the placebo, T-100, and T-200 treatment groups was 0.3 kg (0.4%) , 2.0 kg (3.0%), and 0.9 kg (1.4%), respectively (p = 0.021 for T-100 versu s placebo; p = 0.53 for T-200 versus placebo). Of the 64 patients who compl eted the 8 weeks of study treatment, significant weight gain was observed i n both the T-100 group (2.2 kg, [33%]; p = 0.008 versus placebo) and the T- 200 group (1.5 kg [2.5%]; p = 0.019 versus placebo). Approximately half the weight gain was fat-free mass (bioimpedance analysis). Patients in the T-1 00 or T-200 groups had no significant change in CD4(+) cell counts, neutrop hil counts, or TNF-alpha levels, compared with placebo. HIV viral load meas ured as log(10) copies/ml decreased by a median of 0.07 in the placebo grou p, and increased by a median of 0.29 (T-100 group) and 0.23 (T-200 group) ( p = 0.024 and p = 0.018 versus placebo, respectively). Thalidomide therapy was associated with mild to moderate rashes and fevers, but not peripheral neuropathy. Although the anabolic benefits of high-dose thalidomide are lim ited by drug intolerance, 8 weeks of low-dose thalidomide results in signif icant weight gain in patients with AIDS-associated wasting.