Abstract: Objective In the hospitalized patients with agranulocytosis and persistent fever, the efficacy and safety of domestic caspofungin were further evaluated on the basis of the previous study which comparing the pharmacokinetic characteristics of domestic and original caspofungin for empirical antifungal therapy. Methods This study was a prospective, multi-center, open clinical trial (ClinicalTrials.gov Identifier:NCT03857399). Hospitalized patients with agranulocytosis and persistent fever were enrolled. After the empirical treatment of caspofungin, the efficacy and safety of domestic caspofungin in all patients were evaluated. Results Of the 32 patients enrolled, there were 32 patients in full analysis set (FAS) and 30 patients in Per-protocol Set (PPS). In the FAS and PPS, the clinical efficacy rate of domestic caspofungin were 71.88%(23/32) and 76.67%(23/30), the treatment courses were (13.22±5.80) days and (13.70±5.62) days, respectively. Within 0 to 2 days after treatment, the pyretolysis rate were 78.13%(25/32) and 80%(24/30), and the proportion of patients whose absolute neutrophil count over 0.5×10⁹/L were 75%(24/32) and 76.67%(23/30) in the two set. The 28-day survival rate after treatment were 81.25%(26/32) and 83.33%(25/30), respectively. Domestic caspofungin was well tolerated. Most of the adverse events were grade 1 to grade 2 and drug-unrelated, no adverse events led to dosage decreased or withdrew. The incidence of drug-related adverse events was 3.1%(1/32), including 1 case of hepatic dysfunction. No drug-related serious adverse events occurred. Conclusions Domestic caspofungin had achieved great clinical efficacy and safety as empirical antifungal therapy in patients with agranulocytosis and persistent fever.

Key words: agranulocytosis, fever, domestic caspofungin, clinical efficacy rate, safety