Clinical characteristics, diagnosis, and management of central nervous system aspergillosis in children: a single-center experience.

backgroundCentral nervous system (CNS) aspergillosis is a severe and frequently misdiagnosed infection in pediatric patients. Systematic pediatric data on its clinical, radiological, diagnostic, and therapeutic features remain limited.

methodsWe retrospectively identified children aged 0-18 years with proven or probable CNS aspergillosis admitted to Beijing Children's Hospital between January 2010 and December 2024. Demographic, clinical, laboratory, and imaging data were collected. Treatment regimens and clinical outcomes were systematically evaluated.

resultsSixteen patients were included (12 males), with a median age of 5 years. Hematological malignancies were the most common predisposing factor. Notably, 25% (4/16) of patients lacked identifiable predisposing conditions. Clinical presentations were nonspecific, with fever, seizures, and impaired consciousness being the most common features, and an initial misdiagnosis occurred in 56.3% (9/16) of cases. In contrast to the low yield of cerebrospinal fluid (CSF) cultures, CSF metagenomic next-generation sequencing (mNGS) detected Aspergillus nucleic acids in all tested patients (7/7). Magnetic resonance imaging (MRI) most commonly revealed irregular cerebral abscesses (14/16), frequently accompanied by meningeal enhancement (14/16) and obstructive hydrocephalus (10/16). Among evaluable patients receiving initial voriconazole monotherapy, a partial response was observed in 22.2% (2/9). Conversely, higher response rates were observed with regimens containing liposomal amphotericin B (L-AmB), including initial combination therapy (75.0%) and salvage treatment (80.0%). The all-cause mortality rate was 37.5% (6/16), and moderate-to-severe disability was present in 30.0% (3/10) of survivors.

conclusionsPediatric CNS aspergillosis can occur across a broad risk spectrum, often with nonspecific symptoms, leading to frequent misdiagnosis. Our findings support the early incorporation of CSF mNGS and comprehensive neuroimaging (including whole-neuraxis MRI when clinically indicated) to facilitate timely diagnosis and assess dissemination. The observed high rate of progression with initial voriconazole monotherapy and the relatively favorable responses associated with regimens containing L-AmB highlight the need for prospective pediatric studies to refine initial treatment strategies in severe disease. CLINICAL

trial registrationNot applicable.