SponsorHaikou Affiliated Hospital of Central South University Xiangya School of Medicine
About this trial
This is a single-center, single-arm, observational clinical study evaluating the efficacy and safety of low-dose liposomal amphotericin B (50 mg/day, intravenous, once daily) for the prevention of invasive fungal infections in adult patients (aged 18-75 years) with hematological malignancies who develop prolonged neutropenia (absolute neutrophil count ≤0.5×10\^9/L, expected to last \>7 days) and are at high risk for invasive fungal disease. Participants are those who, per the treating physician's routine clinical decision, have been initiated on liposomal amphotericin B prophylaxis at 50 mg/day due to intolerance or toxicity to other antifungal agents. The primary outcome is the incidence of proven or probable invasive fungal disease. Secondary outcomes include incidence of pneumonia, persistent unexplained fever \>4 days, use of additional systemic antifungal therapy, and adverse events. A total of 30 participants will be enrolled. Data will be collected at baseline, during treatment, and within 7 days after treatment completion.
Eligibility criteria
This trial does not accept healthy volunteers
Qualifiers
Age 18 to 75 years (inclusive), both sexes.
Meets NCCN 2025 V1 guideline criteria for high-risk invasive fungal disease, including allogeneic hematopoietic stem cell transplantation, autologous hematopoietic cell transplantation with mucosal damage, acute leukemia, grade 3/4 graft-versus-host disease, myelodysplastic syndrome, lymphoma, multiple myeloma, chronic lymphocytic leukemia, treatment with purine analogues (fludarabine, clofarabine, nelarabine), chimeric antigen receptor (CAR) T-cell therapy, alemtuzumab therapy, with expected neutropenia >7 days and accompanied by agranulocytosis.
Assessed by the study physician as having high-risk for invasive fungal infection, intolerant or unable to use other antifungal agents due to toxicity or other reasons, and the treating physician has independently decided in routine clinical practice to initiate liposomal amphotericin B for antifungal prophylaxis for 3-5 days, with the selected dosage regimen of 50 mg/day, intravenous, once daily.
Patient or legally authorized representative has voluntarily signed the informed consent form.
Disqualifiers
Allergy to any component of liposomal amphotericin B, or development of serious adverse events during the initial 3-5 days of prophylactic use.
Prior history of proven or probable invasive fungal disease (IFD).
Presence of pneumonia, unexplained fever, or clinical/imaging evidence suggestive of or diagnosed as fungal infection during screening.
Clinically significant hypokalemia (defined as serum potassium <3.2 mmol/L, or below the lower limit of normal while receiving digitalis therapy) that cannot be corrected before starting trial treatment.
Trial population
Adult patients (aged 18-75 years) with hematological malignancies who meet NCCN 2025 V1 high-risk criteria for invasive fungal disease, with expected neutropenia \>7 days (absolute neutrophil count ≤0.5×10⁹/L), who have been initiated on liposomal amphotericin B prophylaxis at 50 mg/day by the treating physician in routine clinical practice due to intolerance or toxicity to other antifungal agents.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Liposomal Amphotericin B
Drug
Liposomal amphotericin B at 50 mg/day, administered intravenously once daily, for antifungal prophylaxis in patients with prolonged neutropenia. Liposomal amphotericin B is a broad-spectrum polyene antifungal agent with activity against most pathogenic fungi, including Candida, Aspergillus, and Mucorales species. The liposomal formulation reduces nephrotoxicity compared to conventional amphotericin B deoxycholate while maintaining equivalent antifungal activity.
Treatment groups
30 Participants
are divided into 1 treatment group
Group A: Liposomal Amphotericin B Prophylaxis Group1 intervention
Trial outcomes
Primary outcomes
1
Incidence of Proven or Probable Invasive Fungal Disease
Proven or probable invasive fungal disease (IFD) diagnosed according to the Sixth Revised Edition of the Diagnostic Criteria and Treatment Principles for Invasive Fungal Disease in Patients with Hematological Malignancies. Proven IFD requires histopathological evidence or positive culture from a sterile site. Probable IFD requires the presence of host factors, clinical features, and mycological evidence.
Time frame
From baseline to 7 days after the end of antifungal prophylaxis treatment
Secondary outcomes
1
Incidence of Pneumonia With No Identified Pathogen
Incidence of pneumonia occurring during the study period with no pathogen identified by microbiological or molecular methods, including but not limited to blood cultures, bronchoalveolar lavage fluid cultures, and NGS.
Time frame
From baseline to 7 days after the end of antifungal prophylaxis treatment
2
Proportion of Participants With Persistent Unexplained Fever >4 Days
Proportion of participants with persistent unexplained fever lasting more than 4 days during the study period. Unexplained fever is defined as fever without an identifiable infectious source after initial clinical evaluation.
Time frame
From baseline to 7 days after the end of antifungal prophylaxis treatment
3
Proportion of Participants Requiring Additional Systemic Antifungal Therapy
Proportion of participants who require systemic antifungal therapy other than the study drug (low-dose liposomal amphotericin B) for suspected or confirmed fungal infection during the study period.
Time frame
From baseline to 7 days after the end of antifungal prophylaxis treatment
4
Discontinuation Rate Due to Adverse Effects or Intolerance
Proportion of participants who discontinue liposomal amphotericin B prophylaxis due to drug-related adverse effects or intolerance during the treatment period.
Time frame
Throughout the treatment period (from Day 1 to the end of liposomal amphotericin B therapy)
Other outcomes
Sponsors and contacts
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