Issues related to the design and interpretation of clinical trials of salvage therapy for invasive mold infection

Nikolaos G. Almyroudis, Dimitrios P. Kontoyiannis, Kent A. Sepkowitz, Ben E. DePauw, Thomas J. Walsh, Brahm H. Segal

Research output: Contribution to journalReview articlepeer-review

Abstract

Invasive mold infection is a major cause of morbidity and mortality among severely immunocompromised individuals. We discuss the challenges involved in the design and interpretation of salvage antifungal trials, focusing on mold infection. We suggest that patients with refractory fungal infection be analyzed separately from those with intolerance to standard regimens because of the poorer prognosis of the former group. We propose a composite outcome assessment in which refractory infection is defined as infection associated with the worsening of at least 2 of the following 3 types of criteria: clinical, radiologic, and mycologic. Confounding variables, including heterogeneity in host factors, initial antifungal therapy, and selection bias, are discussed. Although randomized studies would provide the most credible results, the lack of an adequate number of patients to meet prespecified stratification criteria for all confounding variables makes such studies impractical. Given that randomized studies are unrealistic, studies involving carefully selected, matched, contemporaneous control subjects are likely to be the most useful alternative.

Original languageEnglish (US)
Pages (from-to)1449-1455
Number of pages7
JournalClinical Infectious Diseases
Volume43
Issue number11
DOIs
StatePublished - Dec 1 2006

ASJC Scopus subject areas

  • Microbiology (medical)
  • Infectious Diseases

Fingerprint Dive into the research topics of 'Issues related to the design and interpretation of clinical trials of salvage therapy for invasive mold infection'. Together they form a unique fingerprint.

Cite this