Current and future trials with zidovudine

The first placebo-controlled trial of zidovudine in the management of HIV infection involved patients with the acquired immune deficiency syndrome (AIDS) following their first episode of Pneumocystis carinii pneumonia and patients with severe AIDS-related complex (ARC). Zidovudine was shown to increase survival, and the trial was terminated early at the request of an independent review board. In order to obtain more information on the long-term efficacy and tolerance of the drug the study was continued on an open-label basis where all patients were offered zidovudine. Data from this ongoing open-label study are reviewed on a regular basis. Side-effects associated with zidovudine include anaemia and neutropenia both of which are more predominant in patients with more advanced disease. Two basic strategies have been adopted with the aim of improving the therapeutic index of the drug involving (i) dose modification and (ii) combination with other antiviral or immunomodulatory compounds. Although several phase I and II studies are proceeding it is likely that research in this area will continue to expand. AIDS patients with Kaposi's sarcoma (but without a history of AIDS-defining opportunistic infection) were precluded from the original phase II trial described above. The value of zidovudine in the treatment of the sarcoma per se has yet to be established. Trials are currently in progress in this indication evaluating the potential of zidovudine administered either alone or in combination with interferon. Possibly the largest area of research is concerned with defining the role of zidovudine earlier in the course of HIV infection.(ABSTRACT TRUNCATED AT 250 WORDS)