Objective:To evaluate the efficacy and safety of liposomal amphotericin B (L-AmB) as monotherapy or combination therapy in the treatment of invasive fungal disease (IFD) in children with underlying hematologic diseases.Methods:This study was a cohort study. A retrospective analysis was conducted on clinical data from pediatric patients with underlying hematologic diseases and IFD who were hospitalized in Shanghai Children′s Medical Center, School of Medicine, Shanghai Jiao Tong University between September 2023 and June 2024. The basic information, antifungal treatment status, adverse reactions, and treatment efficacy were collected. Patients were divided into initial treatment group and previously treated group based on whether the duration of previous use of antifungal drugs other than L-AmB during the course of this IFD exceeded seven days. The efficacy and safety of L-AmB, either as monotherapy or combination therapy, were assessed. The influencing factors of all-cause mortality in pediatric patients three months after L-AmB treatment were analyzed using logistic regression.Results:A total of 52 pediatric patients were included in the study, including 23 in the initial treatment group and 29 in the previously treated group. The ages of the patients in the two groups were (8.7±4.6) years and (11.2±3.8) years, respectively. Acute leukemia were the most common underlying hematologic diseases, accounting for 87.0% (20/23) in the initial treatment group and 82.8%(24/29) in the previously treated group. The proportion of underlying hematologic diseases in progressive or relapsed status was 78.3%(18/23) in the initial treatment group and 69.0%(20/29) in the previously treated group. The dosage of L-AmB was (3.65±0.89) mg/(kg·d) in the initial treatment group and (3.56±0.85) mg/(kg·d) in the previously treated group, with treatment duration of 28(13, 40) days and 21(14, 43) days, respectively. Combination therapy was used in 73.9%(17/23) of the initial treatment group and 79.3%(23/29) of the previously treated group. The favorable response rates at the end of L-AmB treatment were 87.0%(20/23) in the initial treatment group and 82.8%(24/29) in the previously treated group. At three months post-treatment, the favorable response rates in these two groups were 65.2%(15/23) and 58.6%(17/29), respectively. Among children with normal baseline serum creatinine levels, the proportion who exhibited an increase in creatinine levels exceeding the upper limit of normal was 4.5%(1/22) in the initial treatment group and 7.7%(2/26) in the previously treated group. Results from logistic regression analysis indicated that children with underlying hematologic diseases in progression or relapse status (odds ratio ( OR)=12.476, 95% confidence interval ( CI) 1.077 to 144.504, P=0.043) and IFD involving at least two non-adjacent organs ( OR=9.006, 95% CI 1.082 to 74.949, P=0.042) were independent risk factors for all-cause mortality three months after L-AmB treatment. Conclusions:Monotherapy or combination therapy with L-AmB demonstrates substantial efficacy and safety in the treatment of IFD in children with underlying hematologic diseases. Progressive or relapsed underlying hematologic diseases and IFD involving at least two non-adjacent organs are independent risk factors for all-cause mortality three months after L-AmB treatment.

Objective:To evaluate the efficacy and safety of liposomal amphotericin B (L-AmB) as monotherapy or combination therapy in the treatment of invasive fungal disease (IFD) in children with underlying hematologic diseases.Methods:This study was a cohort study. A retrospective analysis was conducted on clinical data from pediatric patients with underlying hematologic diseases and IFD who were hospitalized in Shanghai Children′s Medical Center, School of Medicine, Shanghai Jiao Tong University between September 2023 and June 2024. The basic information, antifungal treatment status, adverse reactions, and treatment efficacy were collected. Patients were divided into initial treatment group and previously treated group based on whether the duration of previous use of antifungal drugs other than L-AmB during the course of this IFD exceeded seven days. The efficacy and safety of L-AmB, either as monotherapy or combination therapy, were assessed. The influencing factors of all-cause mortality in pediatric patients three months after L-AmB treatment were analyzed using logistic regression.Results:A total of 52 pediatric patients were included in the study, including 23 in the initial treatment group and 29 in the previously treated group. The ages of the patients in the two groups were (8.7±4.6) years and (11.2±3.8) years, respectively. Acute leukemia were the most common underlying hematologic diseases, accounting for 87.0% (20/23) in the initial treatment group and 82.8%(24/29) in the previously treated group. The proportion of underlying hematologic diseases in progressive or relapsed status was 78.3%(18/23) in the initial treatment group and 69.0%(20/29) in the previously treated group. The dosage of L-AmB was (3.65±0.89) mg/(kg·d) in the initial treatment group and (3.56±0.85) mg/(kg·d) in the previously treated group, with treatment duration of 28(13, 40) days and 21(14, 43) days, respectively. Combination therapy was used in 73.9%(17/23) of the initial treatment group and 79.3%(23/29) of the previously treated group. The favorable response rates at the end of L-AmB treatment were 87.0%(20/23) in the initial treatment group and 82.8%(24/29) in the previously treated group. At three months post-treatment, the favorable response rates in these two groups were 65.2%(15/23) and 58.6%(17/29), respectively. Among children with normal baseline serum creatinine levels, the proportion who exhibited an increase in creatinine levels exceeding the upper limit of normal was 4.5%(1/22) in the initial treatment group and 7.7%(2/26) in the previously treated group. Results from logistic regression analysis indicated that children with underlying hematologic diseases in progression or relapse status (odds ratio ( OR)=12.476, 95% confidence interval ( CI) 1.077 to 144.504, P=0.043) and IFD involving at least two non-adjacent organs ( OR=9.006, 95% CI 1.082 to 74.949, P=0.042) were independent risk factors for all-cause mortality three months after L-AmB treatment. Conclusions:Monotherapy or combination therapy with L-AmB demonstrates substantial efficacy and safety in the treatment of IFD in children with underlying hematologic diseases. Progressive or relapsed underlying hematologic diseases and IFD involving at least two non-adjacent organs are independent risk factors for all-cause mortality three months after L-AmB treatment.