Background. Studies have reported that plasma human immunodeficiency virus type 1 (HIV-1) RNA levels and CD4+ lymphocyte counts in HIV-infected patients improved after treatment with granulocyte-macrophage colony-stimulating factor (GM-CSF). Methods. In AIDS Clinical Trials Group Protocol 5041, 116 patients were enrolled in a double-blind, randomized, placebo-controlled clinical trial of 16 weeks of 250 μg of GM-CSF administered subcutaneously 3 times/week, followed by open-label treatment for an additional 32 weeks. Patients had stable baseline plasma HIV-1 RNA levels of ≥ 1500 copies/mL and received constant antiretroviral regimens through at least the first 16 weeks of the study. Results. After 16 weeks, the GM-CSF group tended to have greater, though clinically insignificant, increases in plasma HIV-1 RNA levels, compared with the placebo group (median change, +0.048 vs. -0.103 log copies/mL; P = .036, in a post hoc analysis). There were trends toward progressive modest increases in CD4+ lymphocyte counts with GM-CSF treatment at 16 weeks (median change, +14 vs. -6 cells/mm3; P = .06) and beyond. Conclusions. GM-CSF does not have an antiviral effect in patients with ongoing HIV replication but may increase CD4+ lymphocyte counts.
Bibliographical noteFunding Information:
Financial support: National Institutes of Health (grants 1U01 AI-46370, AI-38855, 5U01 AI-38855, UO1 AI-25859-17, AI-25903, AI-25924, AI-27660, AI-28691, RR-00865, AI-38858, AI-25879, AI-36219, 5UO1 AI-39156, and 5UO1 AI-32782); National Center for Research Resources.