Objective To analyze the effect of long-term anti-viral treatment in children with acquired immune deficiency syndrome (AIDS) and investigate the factors affecting the treatment efficacy and growth and development of the children, so as to provide reference for improving the efficacy of antiviral drugs. Methods Children with AIDS receiving anti-retroviral treatment during 2004 to 2016 were retrospectively enrolled. The height, weight and CD4+ T cell counts were recorded every half year and the measurement of HIV RNA load was recorded on an annual basis. The CD4+ T cell counts and viral inhibition rates for the children who were under the treatment in the first year, 1~<5 years, 5~<10 years, and ≥10 years were compared. And their growth and development were also assessed. Treatment efficacy and growth and development of the children were compared between those who raised by social organization and by family. Children who raised by family were further divided into two groups: high-income and low-income groups. All categorical data were analyzed using chi-square test and those non-normal distribution were compared by rank-sumtest. Results After comparison between the children who have received anti-virus treatment for 1 to 5 years (including 5 year) and those for 5 to 10 year (including 10 years), the baseline CD4+ T cell counts were 436.5(265, 728)cells/μL and 334 (102, 535)cells/μL, respectively with the statistically significant difference (Z=-2.619, P 0.05); and viral inhibition rates were 92.9% and 97.6%, respectively with no statistical significance (χ2=1.071, P>0.05). The viral inhibition rate for the children receiving the treatment for 1 year was 85.7%, while that for whose treatment lasted for more than 10 years was 100.0%. A total of 5 cases developed drug-resistance (2 cases treated for 1 to 5 years and 3 cases for 5 to 10 years), and the virus replication was completely inhibited after switching to Lopinavir/ritonavir (LPV/r). The drug compliance was more than 95.0%. 64.8% of children met the standard height, while 68.5% met the standard body mass. The baseline and last measured CD4+ T cell counts showed no significant differences between family-raised and social organization-raised children (Z=-1.159 and -0.523, respectively, both P>0.05). The children from high-income families had no significant differences compared with those from low-income ones in terms of the baseline and last measured CD4+ T cell counts (Z=-0.019 and -0.776, respectively, both P>0.05). Conclusions The long-term anti-retroviral treatment can effectively elevate the CD4+ T cell counts, inhibit viral replication and ensure drug compliance, which may promote the growth and development of children. However, approximately 30% children are still lower than the normal standards in terms of height and body mass. The drug-taking observer plays a central role on treatment effect. Most of the children′s family suffer from poor economic conditions. Key words: AIDS, children; Antiretroviral therapy, highly active; Curative effect; Status quo