Surrogate markers for survival in patients with AIDS and AIDS related complex treated with zidovudine

BMJ. 1991 Jan 12;302(6768):73-8. doi: 10.1136/bmj.302.6768.73.

Abstract

Objective: To determine whether early effects of zidovudine treatment on CD4+ lymphocyte count and concentrations of beta 2 microglobulin, neopterin, or HIV p24 antigen or antibody are correlated with survival in patients with AIDS or AIDS related complex.

Design: Retrospective study of changes in laboratory markers and survival.

Setting: Multicentre trial at university hospital clinics.

Subjects: 90 Patients with AIDS or AIDS related complex.

Intervention: Patients started zidovudine 200 mg orally every four hours. Fifty six of the patients died a median 17 months after starting zidovudine; the remaining 34 patients were followed up for a median 25.5 months.

Main outcome measures: Changes in CD4+ lymphocyte count and serum concentrations of p24 antigen and antibody, beta 2 microglobulin, and neopterin; survival of the patient.

Results: The pretreatment characteristics that independently predicted poor survival were determined using a multivariate proportional hazards model: a diagnosis of AIDS (v AIDS related complex), age over 45 years, and the logarithm of serum neopterin concentration. When these baseline characteristics were controlled for the logarithm of CD4+ lymphocyte count at weeks 8-12 of treatment (p = 0.007) and an increase in serum beta 2 microglobulin concentration at weeks 8-12 (p = 0.05) also independently correlated with survival. In the 38 patients with a better pretreatment prognosis, 24 month survival estimated by the product-limit method was 88% for those with a good response on both surrogate markers during early treatment compared with only 50% for those with a poor response on either marker. In the 38 with a worse pretreatment prognosis, 24 month survival was estimated to be 49% for those with a good response on both surrogate markers compared with only 18% for those with a poor response on either.

Conclusion: These data suggest that CD4+ lymphocyte count at 8-12 weeks and, perhaps, change in serum beta 2 microglobulin concentration could be surrogate end points for clinical outcome in trials of antiretroviral drugs for patients with HIV disease.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • AIDS-Related Complex / blood*
  • AIDS-Related Complex / drug therapy
  • Acquired Immunodeficiency Syndrome / blood*
  • Acquired Immunodeficiency Syndrome / drug therapy
  • Acquired Immunodeficiency Syndrome / mortality
  • Adult
  • Biomarkers / blood*
  • Biopterins / analogs & derivatives
  • Biopterins / blood
  • CD4-Positive T-Lymphocytes
  • Female
  • Gene Products, gag / analysis
  • HIV Antibodies / analysis
  • HIV Antigens / analysis
  • HIV Core Protein p24
  • Humans
  • Leukocyte Count
  • Male
  • Middle Aged
  • Neopterin
  • Predictive Value of Tests
  • Prognosis
  • Retrospective Studies
  • Viral Core Proteins / analysis
  • Zidovudine / therapeutic use*
  • beta 2-Microglobulin / metabolism

Substances

  • Biomarkers
  • Gene Products, gag
  • HIV Antibodies
  • HIV Antigens
  • HIV Core Protein p24
  • Viral Core Proteins
  • beta 2-Microglobulin
  • Biopterins
  • Zidovudine
  • Neopterin