Comparison of caspofungin and amphotericin B for invasive candidiasis

N Engl J Med. 2002 Dec 19;347(25):2020-9. doi: 10.1056/NEJMoa021585.


Background: Caspofungin is an echinocandin agent with fungicidal activity against candida species. We performed a double-blind trial to compare caspofungin with amphotericin B deoxycholate for the primary treatment of invasive candidiasis.

Methods: We enrolled patients who had clinical evidence of infection and a positive culture for candida species from blood or another site. Patients were stratified according to the severity of disease, as indicated by the Acute Physiology and Chronic Health Evaluation (APACHE II) score, and the presence or absence of neutropenia and were randomly assigned to receive either caspofungin or amphotericin B. The study was designed to compare the efficacy of caspofungin with that of amphotericin B in patients with invasive candidiasis and in a subgroup with candidemia.

Results: Of the 239 patients enrolled, 224 were included in the modified intention-to-treat analysis. Base-line characteristics, including the percentage of patients with neutropenia and the mean APACHE II score, were similar in the two treatment groups. A modified intention-to-treat analysis showed that the efficacy of caspofungin was similar to that of amphotericin B, with successful outcomes in 73.4 percent of the patients treated with caspofungin and in 61.7 percent of those treated with amphotericin B (difference after adjustment for APACHE II score and neutropenic status, 12.7 percentage points; 95.6 percent confidence interval, -0.7 to 26.0). An analysis of patients who met prespecified criteria for evaluation showed that caspofungin was superior, with a favorable response in 80.7 percent of patients, as compared with 64.9 percent of those who received amphotericin B (difference, 15.4 percentage points; 95.6 percent confidence interval, 1.1 to 29.7). Caspofungin was as effective as amphotericin B in patients who had candidemia, with a favorable response in 71.7 percent and 62.8 percent of patients, respectively (difference, 10.0 percentage points; 95.0 percent confidence interval, -4.5 to 24.5). There were significantly fewer drug-related adverse events in the caspofungin group than in the amphotericin B group.

Conclusions: Caspofungin is at least as effective as amphotericin B for the treatment of invasive candidiasis and, more specifically, candidemia.

Publication types

  • Clinical Trial
  • Comparative Study
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Aged, 80 and over
  • Amphotericin B / adverse effects
  • Amphotericin B / therapeutic use*
  • Anti-Bacterial Agents / adverse effects
  • Anti-Bacterial Agents / therapeutic use*
  • Antifungal Agents / adverse effects
  • Antifungal Agents / therapeutic use*
  • Candida / isolation & purification
  • Candidiasis / classification
  • Candidiasis / complications
  • Candidiasis / drug therapy*
  • Candidiasis / mortality
  • Caspofungin
  • Echinocandins
  • Female
  • Fungemia / drug therapy*
  • Fungemia / mortality
  • Humans
  • Infusions, Intravenous
  • Lipopeptides
  • Male
  • Middle Aged
  • Neutropenia / complications
  • Peptides*
  • Peptides, Cyclic*
  • Recurrence


  • Anti-Bacterial Agents
  • Antifungal Agents
  • Echinocandins
  • Lipopeptides
  • Peptides
  • Peptides, Cyclic
  • Amphotericin B
  • Caspofungin