Efavirenz expanded access program: North American experience

The objective of this study was to make efavirenz (EFV) available to HIV-infected individuals through an expanded access program (EAP) and to obtain additional safety data on EFV. HIV-infected individuals, greater than or equal to 13 years of age, who were either failing or intolerant to their current antiretroviral (ARV) therapy, and who had a CD4 count of less than or equal to 400 cells/mm super(3) were enrolled in the program. Patients received 601) mg EFV once daily in combination with at least one (preferably two) new ARV agent(s). Patients were seen monthly for clinical and safety evaluations. Plasma HIV RNA and C1)4 count reporting was optional. Patients were enrolled from November 1997-March 1999. 8281 patients received EFV. Demographics: 88.5% male; mean age, 40 years; median baseline CD4 count, 135 cells/mm super(3) ( 100 000 copies/ml, 41%); 96% PI-experienced (79%, greater than or equal to 2 PIs); and 47% NNRTI-experienced. At month 6, mean increase in CD4 count from baseline was 37 cells/mm super(3) and mean reduction in plasma HIV RNA was -1.0 log sub(10) copies/ml. Mean reduction in plasma HIV RNA differed significantly when stratified by NNRTI experience: -1.3 versus -0.5 log sub(10) copies/ml for those who were NNRTI-naive or -experienced, respectively. Discontinuations from adverse experiences (AEs), progression of HIV disease, and death were 8.5%, 3%, and 1%, respectively. In conclusion, in heavily ARV-experienced patients, EFV was generally well tolerated, with rates of AE-associated discontinuation similar to those observed in clinical trials. Previous treatment with an NNRTI was associated with lesser reductions in plasma HIV RNA levels.