Zidovudine, didanosine, or both as the initial treatment for symptomatic HIV-infected children

Janet A. Englund, Carol J. Baker, Claire Raskino, Ross E. Mckinney, Barbara Petrie, Mary Glenn Fowler, Deborah Pearson, Anne Gershon, George D. Mcsherry, Elaine J. Abrams, Jenny Schliozberg, John L. Sullivan, Rachel Behrman, James C. Connor, Seth Hetherington, Marta H. Lifschitz, Colin McLaren, Herman Mendez, Karen Millison, Jack MoyeMolly Nozyce, Karen O'Donnell, Lynette Purdue, David Schoenfeld, Gwendolynn Scott, Stephen A. Spector, Diane W. Wara

Research output: Contribution to journalArticlepeer-review

145 Scopus citations


Background: Zidovudine has been the drug of choice for the initial treatment of symptomatic children infected with the human immunodeficiency virus (HIV). This trial was designed to assess the efficacy and safety of treatment with zidovudine alone as compared with either didanosine alone or combination therapy with zidovudine plus didanosine. Methods: In this multicenter, double-blind study, symptomatic HIV-infected children 3 months through 18 years of age were stratified according to age (<30 months or ≤30 months) and randomly assigned to receive zidovudine, didanosine, or zidovudine plus didanosine. The primary end point was length of time to death or to progression of HIV disease. Results: Of the 831 children who could be evaluated, 92 percent had never received antiretroviral therapy and 90 percent had acquired HIV perinatally. An interim analysis (median follow-up, 23 months) showed a significantly higher risk of HIV-disease progression or death in patients receiving zidovudine alone than in those receiving combination therapy (relative risk, 0.61; 95 percent confidence interval, 0.42 to 0.88; P=0.007). The study arm with zidovudine alone was stopped and unblinded; the other two treatment arms were continued. At the end of the study, didanosine alone had an efficacy similar to that of zidovudine plus didanosine (median follow-up, 32 months) (relative risk of disease progression or death, 0.98; 95 percent confidence interval, 0.70 to 1.37; P=0.91). A significantly lower risk of anemia or neutropenia was seen in patients receiving didanosine alone (P=0.036). Conclusions: In symptomatic HIV-infected children, treatment with either didanosine alone or zidovudine plus didanosine was more effective than treatment with zidovudine alone. The efficacy of didanosine alone was similar to that of the combination therapy and was associated with less hematologic toxicity.

Original languageEnglish (US)
Pages (from-to)1704-1712
Number of pages9
JournalNew England Journal of Medicine
Issue number24
StatePublished - 1997

All Science Journal Classification (ASJC) codes

  • General Medicine


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