To investigate the efficacy and safety of caspofungin in treatment of children with hematological diseases complicated with invasive fungal disease (IFD).

From June 2013 to June 2016, a total of 57 children with hematological diseases who were diagnosed as complicated with IFD, and treated by caspofungin in the department of Pediatric Hematology, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University, were chosen as research objects. Their clinical case data were analyzed retrospectively. Chi-square test and t test were applied to compare treatment effective rates of caspofungin between hematological disease children with different diagnostic grades of IFD, treatment effective rates of caspofungin between the first-line and the second-line treatment in each diagnostic grades of IFD, the survival rates of hematological disease children with different diagnostic grades of IFD after 12 weeks of caspofungin treatment, also the courses of caspofungin treatment between hematological disease children with effective and ineffective treatment, as well as treatment effective rates of caspofungin between hematological disease children with different duration of neutropenia. And the safety of caspofungin in treatment of children with hematological diseases complicated with IFD was also evaluated. This study was in line with the requirements of World Medical Association Declaration of Helsinki revised in 2013, and all guardians of these children signed the informed consent forms for treatment by caspofungin.

①Among hematological disease children of this study, 91.2%(52/57) were single site infected with fungi, and pulmonary infection by fungi was the most common (78.9%, 45/57). Among them, 5 cases (8.8%) were proven IFD, 38 cases (66.7%) were probable IFD, and 14 cases (24.5%) were possible IFD. ②The total treatment effective rate of caspofungin in 57 hematological disease children was 86.0%(49/57). Treatment effective rate of caspofungin in probable and possible IFD children were 90.4%(47/52), which were higher than that of 40.0%(2/5) in proven IFD children, and the difference was statistically significant (χ²=9.597, P=0.001). ③Treatment effective rate of caspofungin as the first-line treatment in hematological disease children with proven and probable IFD was 96.7%(29/30), which was higher than that of 76.9%(10/13) in caspofungin as the second-line treatment, and the difference was statistically significant (χ²=4.190, P=0.041). The effective rate of caspofungin as the first-line treatment in hematological disease children with possible IFD was 90.0%(9/10), which was higher than that of 25.0%(1/4) in caspofungin as the second-line treatment, and the difference was statistically significant (χ²=5.915, P=0.015). ④There was no significant difference among proven, probable and possible IFD in the survival rates after 12 weeks of treatment by caspofungin (P>0.05). ⑤The course of treatment in hematological disease children with effective treatment of caspofungin was (16±7) d, which was longer than that in hematological disease children with ineffective treatment (8±6) d, and the difference was statistically significant (t=3.730, P=0.032). ⑥The effective rate of caspofungin between hematological disease children with duration of neutropenia≥14 d was 66.6%, which was significantly lower than that of hematological disease children with duration of neutropenia <14 d (92.9%), and the difference was statistically significant (χ²=6.284, P=0.012). ⑦None of hematological disease children occurred adverse reactions related to the treatment by caspofungin.

Caspofungin has significant curative effect in treatment of hematological diseases complicated with IFD in children, and neutropenia might affect its effect. Caspofungin has no obvious adverse reactions and it can be recommended for the first-line antifungal therapy in hematological disease children complicated with IFD, and its course of treatment would be more than 14 d.