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A pilot study of prophylactic aerosolized amphotericin B in patients at risk for prolonged neutropenia

S E Myers1, S M Devine, R L Topper

  • 1University of Chicago Medical Center, Department of Medicine, Illinois 60637.

Leukemia & Lymphoma
|October 1, 1992
PubMed

Insights

Inhaled amphotericin B is a safe and well-tolerated prophylactic treatment for patients with neutropenia, preventing invasive aspergillosis. This approach does not hinder subsequent intravenous amphotericin B therapy.

Area of Science:

  • Mycology
  • Clinical Pharmacology
  • Infectious Diseases

Background:

  • Invasive aspergillosis poses a significant threat to patients experiencing prolonged neutropenia, leading to high morbidity and mortality.
  • Effective prophylactic strategies are crucial for managing this opportunistic fungal infection in immunocompromised individuals.

Purpose of the Study:

  • To evaluate the safety and tolerability of escalating doses of aerosolized amphotericin B administered via a face mask nebulizer system.
  • To assess the efficacy of inhaled amphotericin B as a prophylactic agent against invasive aspergillosis in high-risk patients.

Main Methods:

  • A phase I clinical trial involving 26 patients (18 transplant recipients, 8 leukemia patients).
  • Administration of escalating doses (5, 10, 15, 20 mg) of aerosolized amphotericin B twice daily via nebulizer.
  • Monitoring for side effects and clinical outcomes, including the development of invasive aspergillosis and the need for empirical intravenous therapy.

Main Results:

  • Aerosolized amphotericin B was well tolerated at all tested dose levels, with no observed side effects.
  • No patients in the study developed clinically suspected or pathologically confirmed invasive aspergillosis.
  • Prophylactic inhaled therapy did not appear to compromise the effectiveness of subsequent intravenous amphotericin B treatment.

Conclusions:

  • Prophylactic aerosolized amphotericin B is safe and well-tolerated in patients with prolonged neutropenia at doses up to 20 mg twice daily.
  • Inhaled amphotericin B shows promise as an effective prophylactic agent against invasive aspergillosis.
  • Further large-scale, controlled trials are warranted to confirm the efficacy and optimal use of prophylactic inhaled amphotericin B.

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