Analysis of compassionate use itraconazole therapy for invasive aspergillosis by the NIAID Mycoses Study Group criteria.

BACKGROUND: Successful therapy of invasive aspergillosis is difficult, and the place of new drugs is evolving. Earlier studies, with fewer patients, suggest itraconazole, an oral azole, is effective for some patients.
METHODS: Compassionate use data were analyzed by criteria applied previously in a multicenter trial as a reference point. The course of 125 patients was evaluated and their clinical settings and responses were categorized.
RESULTS: Overall, 34 (27%) had a complete response, 45 (36%) improved, 20 (16%) were unchanged, and 26 (21%) worsened. The subset receiving less than 2 weeks of itraconazole therapy had a worse outcome than the remainder of the group as did patients with sinus, central nervous system, or widely disseminated disease. Prior therapy, age, underlying disease, other sites of aspergillosis, dose, or Aspergillus species did not correlate closely with outcome. In patients who responded, a period of months was commonly required before objective improvement was documented. Patients who underwent bone marrow transplantation fared better than in previous reports.
CONCLUSIONS: Itraconazole is effective in many patients with aspergillosis. This large series supports earlier conclusions that response rates are similar to those reported for amphotericin B.