Background:  In the United States, it is routine to treat all infants and children with perinatally acquired HIV infection because of the high risk of rapid disease progression in this population and the inability to identify those destined to be non-progressors. Some older children who discontinue ART do not experience HIV disease progression, but there is no systematic approach to identifying such children for a trial off therapy. We assessed the frequency and characteristics of children who appeared to safely discontinue ART in the Adolescent Master Protocol (AMP) of the Pediatric HIV/AIDS Cohort Study (PHACS), a prospective cohort study designed to determine outcomes of children with perinatally-acquired HIV.  

Methods:  PHACS AMP enrolled 7- to 16-year-old children with perinatally acquired HIV infection at 12 sites, beginning in March 2007. Within this study we identified children who were not receiving ART, had not received ART for ≥6 months, maintained CD4 ≥20% and >350 cells/mm³, and had not progressed to AIDS (Centers for Disease Control and Prevention [CDC] clinical stage C) over their lifetime. Demographics and clinical characteristics of these children, defined as non-progressors, were summarized. 

Results:  Of the 240 children with complete ART history enrolled in PHACS AMP as of August 2008, 12 (5%) were not receiving ART for ≥6 months, 8 (3%) of whom met the CD4 and clinical criteria for non-progressors (see the table). The median age for these 8 children was 14.5 years, 5 (63%) were female, 5 (63%) were black, and 6 (75%) were CDC category N/A. All had previously received ART but subjects 1 to 5 (63%) had never received HAART. Median age at ART initiation was 2.2 years and median duration on ART was 8.8 years. The current duration since discontinuation of all ART ranged from 0.6 to 5.2 years (median 4.0).

Conclusions:  This report identifies a small proportion of non-progressors, despite ART discontinuation among children who survived many years with perinatal HIV infection. The vast majority of children with perinatally acquired HIV infection are receiving ART, but there were children who have never experienced severe disease or immunosuppression despite periods of 6 months to 5 years without ART. It is not known whether early or intermittent ART modified their disease progression. Research is needed to develop a systematic approach to identify perinatally HIV-infected children who may be candidates to discontinue ART.