This is a single-center, single-arm, observational clinical study evaluating the efficacy and safety of low-dose liposomal amphotericin B (1 mg/kg/day, intravenous, once daily) for the prevention of invasive fungal infections in children aged 3-17 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 1 mg/kg/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.
Age 3 to 17 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(a), multiple myeloma(a), chronic lymphocytic leukemia(a), treatment with purine analogues (fludarabine, clofarabine, nelarabine), chimeric antigen receptor (CAR) T-cell therapy, alemtuzumab therapy, with expected neutropenia >7 days(b) 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 1 mg/kg/day, intravenous, once daily.
Patient or legally authorized representative has voluntarily signed the informed consent form.
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.
Children aged 3-17 years with thalassemia or other hematological conditions who undergo allogeneic hematopoietic stem cell transplantation or other intensive therapies associated with prolonged neutropenia (\>7 days), meet NCCN 2025 V1 high-risk criteria for invasive fungal disease, and have been initiated on liposomal amphotericin B prophylaxis at 1 mg/kg/day by the treating physician in routine clinical practice due to intolerance or toxicity to other antifungal agents.
Cohort
Prospective
Liposomal Amphotericin B (LAmB)
DrugLiposomal amphotericin B at 1 mg/kg/day, administered intravenously once daily, for antifungal prophylaxis in children 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.
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.
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.
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.
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.
Discontinuation Rate of Liposomal Amphotericin B 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.
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