To characterize the efficacy and tolerability of multiple drug therapy (MDT) among heavily pre-treated HIV-infected children.

An observational study of seven children treated with 4–7 antiretroviral agents. MDT regimens were chosen with regard to past antiretroviral exposure and genotypic resistance data. Five children received MDT once, one child twice and one child four times. All patients had AIDS and severe CD4⁺ depletion and failed >2 PI-based HAART regimens.

Virologic response, defined as a $\ge {\text{log}}_{10}$ decrease in plasma HIV-1 RNA at week 24, was achieved in 7/11 MDT. Successful MDT kept a sustained viral suppression (<50copies/ml) at longest follow-up (72–96 weeks). Successful MDT obtained a great immune recovery: the median rise in absolute and percentage of CD4⁺ cells was 261 and 4 at week 24 and it reached 480 and 16 at 72–96 weeks. Adverse events were common but generally manageable. Mild/moderate gastrointestinal complaints and laboratory abnormalities were detected in 5/11 and 8/11 MDT. Grade 2 severity pancreatitis occurred in one case with chronic active hepatitis C. Pancreatitis resolved within 30 days of MDT interruption.

MDT may be a therapeutic option in children who failed to respond to most standard HAART regimens.