Isavuconazole provides effective and tolerable antifungal prophylaxis for patients with acute myeloid leukemia

Cameron J Hunter1, Andrew Chou2, Heidi Roeder3

  • 1Section of Medical Oncology and Hematology, Department of Internal Medicine, Yale School of Medicine and Yale Cancer Center, New Haven, CT, USA.

Leukemia Research
|December 9, 2025
PubMed

Insights

Isavuconazole offers a better side-effect profile than voriconazole for antifungal prophylaxis in acute myeloid leukemia (AML) patients. Both agents showed comparable efficacy in preventing invasive fungal infections (IFIs).

Area of Science:

  • Hematology
  • Infectious Diseases
  • Pharmacology

Background:

  • Voriconazole is a standard antifungal prophylactic (AFP) agent for acute myeloid leukemia (AML) patients during neutropenia.
  • Voriconazole use is limited by side-effects like transaminase elevations, visual disturbances, and drug-drug interactions (DDIs).
  • Isavuconazole presents an alternative AFP with a potentially more favorable side-effect profile.

Purpose of the Study:

  • To compare complication rates leading to AFP withdrawal between isavuconazole and voriconazole in AML patients.
  • To evaluate and compare the incidence of invasive fungal infections (IFIs) and overall survival (OS) between the two azole agents.
  • To assess the safety and efficacy of isavuconazole as an antifungal prophylactic option in AML.

Main Methods:

  • Retrospective analysis comparing isavuconazole (n=63) and voriconazole (n=215) for initial AFP in AML patients.
  • Comparison of AFP discontinuation rates due to adverse events or other reasons.
  • Analysis of IFI rates, breakthrough IFI (bIFI), IFI-free survival (IFI-FS), and median overall survival (OS) in subgroups using only isavuconazole (n=41) or voriconazole (n=90).

Main Results:

  • Voriconazole was associated with significantly higher rates of AFP discontinuation due to transaminase elevations (p=0.019), visual disturbances (p=0.002), and DDIs (p=0.002).
  • Isavuconazole was more frequently discontinued due to coverage denial or high cost (p<0.0001).
  • Rates of IFI, bIFI, IFI-FS, and OS were comparable between the two azole agents.

Conclusions:

  • Isavuconazole demonstrates an improved side-effect profile compared to voriconazole for antifungal prophylaxis in AML patients.
  • Isavuconazole is an effective AFP agent with comparable efficacy to voriconazole in preventing IFIs.
  • Isavuconazole should be considered a promising alternative AFP option for AML patients, balancing efficacy and tolerability.

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