What question did this study set out to answer?

To evaluate the efficacy and safety of isavuconazole in patients with invasive mold disease and hematological malignancies.

July 8, 2026Open Access

Isavuconazole for invasive mold disease in patients with hematological malignancies: a multicenter real-world study from China on efficacy, safety, and competing risks

Key Points

To evaluate the efficacy and safety of isavuconazole in patients with invasive mold disease and hematological malignancies.
Retrospective analysis of adult patients with hematological malignancies treated with isavuconazole for ≥72 hours from two Chinese hospitals.
Primary endpoint was week 6 treatment success defined as survival, clinical/radiological improvement, and non-discontinuation due to failure/toxicity.
Secondary endpoints included analysis of all-cause and IMD-attributable mortality at weeks 6 and 12.
Overall week 6 treatment success was 59.5% (50/84), with significant variances across infection types (P=0.007).
Week 12 all-cause mortality rate was 21.6%, with lower IMD-attributable mortality in monotherapy (6.6%) compared to combination therapy (26.1%; P=0.023).
Competing risk analysis showed week 12 attributable mortality rates varied by infection type: aspergillosis (9.7%), mucormycosis (36.0%), and mixed infections (13.0%).

Abstract

ABSTRACT Isavuconazole is approved for invasive aspergillosis (IA) and mucormycosis, but real-world data in patients with hematological malignancies are limited. This retrospective study (2022–2025) included adult patients with hematological malignancies and proven/probable invasive mold disease (IMD) who were treated with isavuconazole for ≥72 h at two Chinese hospitals. The primary endpoint was week 6 treatment success (survival, clinical/radiological improvement, and no discontinuation due to failure/toxicity). Secondary endpoints included all-cause and IMD-attributable mortality at weeks 6 and 12 (competing risk analysis). Among 84 patients, 62 had aspergillosis (69.4% treatment success), 14 had mucormycosis (35.7%), and 8 had mixed infection (25.0%); the overall week 6 treatment success was 59.5% (50/84), with significant variation across infection types ( P = 0.007) and treatment strategy (monotherapy 65.6% vs. combination 43.5%; P < 0.001), but not by therapy role. Week 6 all-cause and IMD-attributable mortality rates were 17.9% and 11.9%, respectively; IMD-attributable mortality rate was lower with monotherapy (6.6%) than with combination therapy (26.1%; P = 0.023). Week 12 all-cause mortality rate was 21.6%. Competing risk analysis showed significant differences in week 12 attributable mortality by infection type (overall P = 0.04): aspergillosis, 9.7%; mucormycosis, 36.0%; and mixed, 13.0%. Monotherapy was associated with lower attributable mortality than combination therapy (8.2% vs. 31.0%; P = 0.01). Four patients (4.8%) had drug-related adverse events (one discontinuation due to vomiting). In this real-world Chinese cohort, isavuconazole achieved 59.5% treatment success with favorable safety. Infection type and treatment strategy were key prognostic factors: aspergillosis and monotherapy were associated with better outcomes, whereas mucormycosis and combination therapy were associated with higher attributable mortality risk.

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