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

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.