Li Zhang,1 Qinglan Zhang,2 Wei Ma,3 Zhang-bo Chu,4 Li Xu,5 Hongyun Xing,6 Jishi Wang,7 Jing Lin,1,8 Zhengyin Liu1 

1Department of Infectious Disease, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing, People’s Republic of China; 2Department of Hematology, Henan Cancer Hospital, Affiliated Cancer Hospital of Zhengzhou University, Zhengzhou, Henan, People’s Republic of China; 3Department of Pediatric Intensive Care Medicine, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, People’s Republic of China; 4Department of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, Hubei, People’s Republic of China; 5Department of Hematology, Anhui No.2 Provincial People’s Hospital, Hefei, Anhui, People’s Republic of China; 6Department of Hematology, the Affiliated Hospital of Southwest Medical University, Luzhou, Sichuan, People’s Republic of China; 7Department of Hematology, the Affiliated Hospital of Guizhou Medical University, Guiyang, Guizhou, People’s Republic of China; 8Graduate School, Peking Union Medical College, Chinese Academy of Medical Sciences, Beijing, People’s Republic of China

Correspondence: Zhengyin Liu, Department of Infectious Disease, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, No. 1 Shuaifuyuan, Dongcheng District, Beijing, 100730, People’s Republic of China, Tel +86-010-69156114, Email zhengyinl@hotmail.com

Purpose: This study aimed to explore the efficacy and safety of amphotericin B colloidal dispersion (ABCD) in treating patients with invasive fungal disease (IFD) or febrile neutropenia.
Patients and Methods: This study retrospectively included patients diagnosed with IFD or febrile neutropenia who received ABCD treatment from 22 hospitals. The efficacy and safety were evaluated at the end of ABCD treatment. The characteristics of patients diagnosed with possible, probable and proven IFD according to the revised EORTC-MSG criteria were then further analyzed to conduct sensitivity analysis.
Results: A total of 503 patients were enrolled in this study. Of these patients, 391 received ABCD treatment for a minimum of seven days, and the overall efficacy of ABCD was determined to be 71.87% (281/391). 183 patients were diagnosed with possible, probable and proven IFD, the ABCD efficacy of whom was 67.76% (124/183). The efficacy of ABCD in patients with hematologic malignancies, AIDS and diabetes was 70.10% (211/301), 88.24% (30/34), and 83.33% (10/12), respectively. In terms of various fungal species, the efficacy of ABCD in patients with Aspergillus, Mucorales and Candida infections was 62.96% (34/54), 80.49% (33/41) and 66.67% (22/33), respectively. For patients in the targeted therapy, diagnostic-driven therapy, and empirical therapy groups, the efficacy of ABCD was 76.92% (60/78), 70.17% (207/295), and 77.78% (14/18), respectively. The most common adverse events (AEs) were infusion reactions (30.22%, 152/503) and hypokalemia (38.97%, 196/503), with the majority of these AEs classified as grade 1– 2. This study was registered with ClinicalTrials.gov, NCT05116059.
Conclusion: ABCD has demonstrated satisfactory therapeutic efficacy and safety in the treatment of IFD or febrile neutropenia.

Keywords: amphotericin B colloidal dispersion, invasive fungal disease, antifungal therapy, febrile neutropenia