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  • AZT Trial Original

    • AZT Trial

    "Azidothymidine (AZT) in Patients with AIDS or Advanced AIDS-Related Complex". The New England Journal of Medicine. 1987. 317(4):185–191. PubMed • Full text • PDF

    Clinical Question


    Does Azidothymidine (AZT) decrease mortality and frequency of opportunistic infections in patients with AIDS or advanced AIDS-related complex (ARC)?

    Bottom Line


    Azidothymidine (AZT) demonstrated a reduction in mortality and opportunistic infections in a selected group of subjects with AIDS or advanced AIDS-related complex, over the 8 to 24 weeks of observation in this study.

    Major Points


    The study established the efficacy of AZT in reducing mortality and morbidity among patients with AIDS or advanced AIDS-related complex. AZT was associated with improvements in clinical status, CD4 cell counts, skin-test reactivity, and p24 antigen levels.

    Guidelines


    The study findings led to the establishment of AZT as a treatment option for patients with AIDS or advanced AIDS-related complex. Subsequent treatment guidelines for HIV/AIDS would consider these findings in their recommendations.

    Design


    - Double-blind, placebo-controlled trial
    - N=282 patients with AIDS manifested by Pneumocystis carinii pneumonia or with advanced AIDS-related complex
    - Intervention: 250 mg of AZT orally every 4 hours
    - Duration: 24 weeks
    - Outcomes measured included mortality, opportunistic infections, clinical status, and immunological markers

    Population


    - Inclusion Criteria: Patients with AIDS and a first episode of P. carinii pneumonia confirmed within the preceding 120 days, or patients with advanced ARC
    - Exclusion Criteria: Multiple opportunistic infections, neoplasm, hemoglobin <9.5 g/dL, absolute CD4 count >500 cells/mm³, recent antiretroviral therapy, and other specified conditions
    - Baseline Characteristics: Comparable age, weight, performance status, symptoms, and CD4 cells between AZT and placebo groups

    Interventions


    - Participants randomized to receive either AZT or placebo
    - Criteria for response: Survival, occurrence of opportunistic infections, Karnofsky performance score, stable weight, improved number of CD4 cells

    Outcomes


    - Primary Outcomes: Mortality and occurrence of opportunistic infections
    - Secondary Outcomes: Karnofsky performance status, weight changes, CD4 cell counts, skin-test reactivity, serum p24 antigen levels

    Criticisms


    - Early termination limited long-term safety and efficacy conclusions.
    - The study did not assess the effectiveness of AZT beyond 24 weeks and larger patient subsets to confirm generalizability.
    - There were concerns regarding the cumulative toxicity of AZT and durability of immunological benefits.

    Funding


    - The study was funded by the Burroughs Wellcome Co., the manufacturer of AZT.

    Further Reading


    - Additional studies and trials have since been conducted to assess the long-term benefits and optimal dosing of AZT, which have further informed treatment guidelines and regimens for HIV/AIDS.
    - Consideration of AZT's role in combination antiretroviral therapy (cART) and the subsequent evolution of HIV treatment strategies.